Gene therapy for childhood immunological diseases View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2008-01

AUTHORS

D B Kohn

ABSTRACT

Gene therapy using autologous hematopoietic stem cells (HSC) that are corrected with the normal gene may have a beneficial effect on blood cell production or function, without the immunologic complications of allogeneic HSC transplantation. Childhood immunological diseases are highly favorable candidates for responses to gene therapy using HSC. Hemoglobinopathies, lysosomal and metabolic disorders and defects of hematopoietic stem and progenitor cells should also be ameliorated by gene therapy using autologous HSC. At present, gene therapy has been beneficial for patients with XSCID, ADA-deficient SCID and chronic granulomatous disease. The principle that partial marrow conditioning increases engraftment of gene-corrected HSC has been demonstrated. Clinical trials are being developed in Europe and the United States to treat several other genetic blood cell disorders. This progress is tempered by the serious complication observed in XSCID patients developing T lymphoproliferative disease. New methods for gene transfer (lentiviral and foamy viral vectors, semi-viral systems and gene correction) may retain or further increase the efficacy and decrease the risks from gene therapy using HSC. Ultimately, the relative benefits and risks of autologous gene therapy will be weighed against other available options (for example, allogeneic HSCT) to determine the treatment of choice. More... »

PAGES

1705895

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/sj.bmt.1705895

DOI

http://dx.doi.org/10.1038/sj.bmt.1705895

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1043623845

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/17994122


Indexing Status Check whether this publication has been indexed by Scopus and Web Of Science using the SN Indexing Status Tool
Incoming Citations Browse incoming citations for this publication using opencitations.net

JSON-LD is the canonical representation for SciGraph data.

TIP: You can open this SciGraph record using an external JSON-LD service: JSON-LD Playground Google SDTT

[
  {
    "@context": "https://springernature.github.io/scigraph/jsonld/sgcontext.json", 
    "about": [
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/1004", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "name": "Medical Biotechnology", 
        "type": "DefinedTerm"
      }, 
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/10", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "name": "Technology", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Antigens, CD34", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Child, Preschool", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Clinical Trials as Topic", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Genetic Therapy", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Hematopoietic Stem Cell Transplantation", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Humans", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Immunologic Deficiency Syndromes", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Infant", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Transplantation, Homologous", 
        "type": "DefinedTerm"
      }
    ], 
    "author": [
      {
        "affiliation": {
          "alternateName": "University of Southern California", 
          "id": "https://www.grid.ac/institutes/grid.42505.36", 
          "name": [
            "Division of Research Immunology/Bone Marrow Transplantation, Department of Pediatrics, Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA", 
            "Department of Molecular Microbiology and Immunology, University of Southern California Keck School of Medicine, Los Angeles, CA, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Kohn", 
        "givenName": "D B", 
        "id": "sg:person.01231045607.82", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01231045607.82"
        ], 
        "type": "Person"
      }
    ], 
    "citation": [
      {
        "id": "https://doi.org/10.1006/mthe.2000.0034", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1005421718"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0140-6736(68)92673-1", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1007159919"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0140-6736(68)92673-1", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1007159919"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm0896-876", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1010628157", 
          "https://doi.org/10.1038/nm0896-876"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/j.ymthe.2006.06.007", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1010867996"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1073/pnas.0608138103", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1011766231"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1056/nejm199902183400703", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012177588"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1128/mcb.21.12.3926-3934.2001", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012411175"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0140-6736(03)12513-5", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012975233"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm1095-1017", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1013283766", 
          "https://doi.org/10.1038/nm1095-1017"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/j.clim.2005.07.006", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1013539255"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1182/blood-2003-05-1558", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1013720305"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1073/pnas.122131099", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1014781978"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0065-2660(05)54009-4", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1016961895"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0065-2660(05)54009-4", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1016961895"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1001/jama.295.5.508", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1016979749"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.1088547", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1018519577"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.288.5466.669", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1019730668"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0140-6736(04)17590-9", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1020712301"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/35017565", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1021691346", 
          "https://doi.org/10.1038/35017565"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/35017565", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1021691346", 
          "https://doi.org/10.1038/35017565"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1182/blood-2006-03-006908", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1022143644"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1172/jci31666", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1023711644"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.1083413", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1024101746"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm0502-423", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1026599498", 
          "https://doi.org/10.1038/nm0502-423"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm0502-423", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1026599498", 
          "https://doi.org/10.1038/nm0502-423"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1172/jci32949", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1028986816"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1172/jci31661", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1035515942"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nature03556", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1040117781", 
          "https://doi.org/10.1038/nature03556"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nature03556", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1040117781", 
          "https://doi.org/10.1038/nature03556"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1172/jci31659", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1042616999"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm0798-775", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1042866864", 
          "https://doi.org/10.1038/nm0798-775"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm1393", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1043834387", 
          "https://doi.org/10.1038/nm1393"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nm1393", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1043834387", 
          "https://doi.org/10.1038/nm1393"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1056/nejmoa012616", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1044007615"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/j.exphem.2006.05.023", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1049719462"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1182/blood-2004-01-0045", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1051628326"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nbt1216", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1053690685", 
          "https://doi.org/10.1038/nbt1216"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nbt1216", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1053690685", 
          "https://doi.org/10.1038/nbt1216"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1089/hum.1990.1.3-257", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1059271001"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.1070104", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1062446366"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.1091667", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1062449108"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.270.5235.470", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1062551476"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.270.5235.475", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1062551477"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.272.5259.263", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1062552729"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://app.dimensions.ai/details/publication/pub.1082981291", 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://app.dimensions.ai/details/publication/pub.1083034885", 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://app.dimensions.ai/details/publication/pub.1083191032", 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://app.dimensions.ai/details/publication/pub.1083254987", 
        "type": "CreativeWork"
      }
    ], 
    "datePublished": "2008-01", 
    "datePublishedReg": "2008-01-01", 
    "description": "Gene therapy using autologous hematopoietic stem cells (HSC) that are corrected with the normal gene may have a beneficial effect on blood cell production or function, without the immunologic complications of allogeneic HSC transplantation. Childhood immunological diseases are highly favorable candidates for responses to gene therapy using HSC. Hemoglobinopathies, lysosomal and metabolic disorders and defects of hematopoietic stem and progenitor cells should also be ameliorated by gene therapy using autologous HSC. At present, gene therapy has been beneficial for patients with XSCID, ADA-deficient SCID and chronic granulomatous disease. The principle that partial marrow conditioning increases engraftment of gene-corrected HSC has been demonstrated. Clinical trials are being developed in Europe and the United States to treat several other genetic blood cell disorders. This progress is tempered by the serious complication observed in XSCID patients developing T lymphoproliferative disease. New methods for gene transfer (lentiviral and foamy viral vectors, semi-viral systems and gene correction) may retain or further increase the efficacy and decrease the risks from gene therapy using HSC. Ultimately, the relative benefits and risks of autologous gene therapy will be weighed against other available options (for example, allogeneic HSCT) to determine the treatment of choice.", 
    "genre": "research_article", 
    "id": "sg:pub.10.1038/sj.bmt.1705895", 
    "inLanguage": [
      "en"
    ], 
    "isAccessibleForFree": true, 
    "isPartOf": [
      {
        "id": "sg:journal.1096890", 
        "issn": [
          "0268-3369", 
          "1476-5365"
        ], 
        "name": "Bone Marrow Transplantation", 
        "type": "Periodical"
      }, 
      {
        "issueNumber": "2", 
        "type": "PublicationIssue"
      }, 
      {
        "type": "PublicationVolume", 
        "volumeNumber": "41"
      }
    ], 
    "name": "Gene therapy for childhood immunological diseases", 
    "pagination": "1705895", 
    "productId": [
      {
        "name": "readcube_id", 
        "type": "PropertyValue", 
        "value": [
          "cfcba49d0c18951ef93c5a63cd783cdd63e4ac89bfe7864326addc8077a9b5d4"
        ]
      }, 
      {
        "name": "pubmed_id", 
        "type": "PropertyValue", 
        "value": [
          "17994122"
        ]
      }, 
      {
        "name": "nlm_unique_id", 
        "type": "PropertyValue", 
        "value": [
          "8702459"
        ]
      }, 
      {
        "name": "doi", 
        "type": "PropertyValue", 
        "value": [
          "10.1038/sj.bmt.1705895"
        ]
      }, 
      {
        "name": "dimensions_id", 
        "type": "PropertyValue", 
        "value": [
          "pub.1043623845"
        ]
      }
    ], 
    "sameAs": [
      "https://doi.org/10.1038/sj.bmt.1705895", 
      "https://app.dimensions.ai/details/publication/pub.1043623845"
    ], 
    "sdDataset": "articles", 
    "sdDatePublished": "2019-04-11T01:47", 
    "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
    "sdPublisher": {
      "name": "Springer Nature - SN SciGraph project", 
      "type": "Organization"
    }, 
    "sdSource": "s3://com-uberresearch-data-dimensions-target-20181106-alternative/cleanup/v134/2549eaecd7973599484d7c17b260dba0a4ecb94b/merge/v9/a6c9fde33151104705d4d7ff012ea9563521a3ce/jats-lookup/v90/0000000001_0000000264/records_8700_00000426.jsonl", 
    "type": "ScholarlyArticle", 
    "url": "http://www.nature.com/articles/1705895"
  }
]
 

Download the RDF metadata as:  json-ld nt turtle xml License info

HOW TO GET THIS DATA PROGRAMMATICALLY:

JSON-LD is a popular format for linked data which is fully compatible with JSON.

curl -H 'Accept: application/ld+json' 'https://scigraph.springernature.com/pub.10.1038/sj.bmt.1705895'

N-Triples is a line-based linked data format ideal for batch operations.

curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/pub.10.1038/sj.bmt.1705895'

Turtle is a human-readable linked data format.

curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1038/sj.bmt.1705895'

RDF/XML is a standard XML format for linked data.

curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1038/sj.bmt.1705895'


 

This table displays all metadata directly associated to this object as RDF triples.

236 TRIPLES      21 PREDICATES      80 URIs      30 LITERALS      18 BLANK NODES

Subject Predicate Object
1 sg:pub.10.1038/sj.bmt.1705895 schema:about N0e4017affd9b4c43a54d4a8ed25b2cf1
2 N1617f63044a842b29810405e21342d93
3 N1682579dda9448cb9b69486feacf31b0
4 N1ccdfbe4f24b49ed96f6bb80b1467fe2
5 N2ba5a3d3aae64d1a8949f293d421c99d
6 N2c7b28fdcc1a44a3a44c551b9cc916e3
7 N362a3dc80c2344b198e18823a65c9b43
8 Nd8f11bf9781f424bb0cd42c81cbe93bb
9 Nfb5294aef777446085a729ab98c7a6cc
10 anzsrc-for:10
11 anzsrc-for:1004
12 schema:author N4348c7e7374a4c9c9468fa56454490ad
13 schema:citation sg:pub.10.1038/35017565
14 sg:pub.10.1038/nature03556
15 sg:pub.10.1038/nbt1216
16 sg:pub.10.1038/nm0502-423
17 sg:pub.10.1038/nm0798-775
18 sg:pub.10.1038/nm0896-876
19 sg:pub.10.1038/nm1095-1017
20 sg:pub.10.1038/nm1393
21 https://app.dimensions.ai/details/publication/pub.1082981291
22 https://app.dimensions.ai/details/publication/pub.1083034885
23 https://app.dimensions.ai/details/publication/pub.1083191032
24 https://app.dimensions.ai/details/publication/pub.1083254987
25 https://doi.org/10.1001/jama.295.5.508
26 https://doi.org/10.1006/mthe.2000.0034
27 https://doi.org/10.1016/j.clim.2005.07.006
28 https://doi.org/10.1016/j.exphem.2006.05.023
29 https://doi.org/10.1016/j.ymthe.2006.06.007
30 https://doi.org/10.1016/s0065-2660(05)54009-4
31 https://doi.org/10.1016/s0140-6736(03)12513-5
32 https://doi.org/10.1016/s0140-6736(04)17590-9
33 https://doi.org/10.1016/s0140-6736(68)92673-1
34 https://doi.org/10.1056/nejm199902183400703
35 https://doi.org/10.1056/nejmoa012616
36 https://doi.org/10.1073/pnas.0608138103
37 https://doi.org/10.1073/pnas.122131099
38 https://doi.org/10.1089/hum.1990.1.3-257
39 https://doi.org/10.1126/science.1070104
40 https://doi.org/10.1126/science.1083413
41 https://doi.org/10.1126/science.1088547
42 https://doi.org/10.1126/science.1091667
43 https://doi.org/10.1126/science.270.5235.470
44 https://doi.org/10.1126/science.270.5235.475
45 https://doi.org/10.1126/science.272.5259.263
46 https://doi.org/10.1126/science.288.5466.669
47 https://doi.org/10.1128/mcb.21.12.3926-3934.2001
48 https://doi.org/10.1172/jci31659
49 https://doi.org/10.1172/jci31661
50 https://doi.org/10.1172/jci31666
51 https://doi.org/10.1172/jci32949
52 https://doi.org/10.1182/blood-2003-05-1558
53 https://doi.org/10.1182/blood-2004-01-0045
54 https://doi.org/10.1182/blood-2006-03-006908
55 schema:datePublished 2008-01
56 schema:datePublishedReg 2008-01-01
57 schema:description Gene therapy using autologous hematopoietic stem cells (HSC) that are corrected with the normal gene may have a beneficial effect on blood cell production or function, without the immunologic complications of allogeneic HSC transplantation. Childhood immunological diseases are highly favorable candidates for responses to gene therapy using HSC. Hemoglobinopathies, lysosomal and metabolic disorders and defects of hematopoietic stem and progenitor cells should also be ameliorated by gene therapy using autologous HSC. At present, gene therapy has been beneficial for patients with XSCID, ADA-deficient SCID and chronic granulomatous disease. The principle that partial marrow conditioning increases engraftment of gene-corrected HSC has been demonstrated. Clinical trials are being developed in Europe and the United States to treat several other genetic blood cell disorders. This progress is tempered by the serious complication observed in XSCID patients developing T lymphoproliferative disease. New methods for gene transfer (lentiviral and foamy viral vectors, semi-viral systems and gene correction) may retain or further increase the efficacy and decrease the risks from gene therapy using HSC. Ultimately, the relative benefits and risks of autologous gene therapy will be weighed against other available options (for example, allogeneic HSCT) to determine the treatment of choice.
58 schema:genre research_article
59 schema:inLanguage en
60 schema:isAccessibleForFree true
61 schema:isPartOf N46dccfae47e044f7a4c6ee85279901fb
62 Nf84f2bb9b53244638388f75d9c55d0db
63 sg:journal.1096890
64 schema:name Gene therapy for childhood immunological diseases
65 schema:pagination 1705895
66 schema:productId N0eb3538e00014a0580d03910a6937d5d
67 N19c161f3da774c3b969f81913b486967
68 N38759fa5c1704c998542d81bf4be8ece
69 N3ef04a951f914e88b9a07150b48bb419
70 Nc1ca8cdfd793470aa0c6bbe4d132d2f4
71 schema:sameAs https://app.dimensions.ai/details/publication/pub.1043623845
72 https://doi.org/10.1038/sj.bmt.1705895
73 schema:sdDatePublished 2019-04-11T01:47
74 schema:sdLicense https://scigraph.springernature.com/explorer/license/
75 schema:sdPublisher Nec86e8b553f949a6b73ee7a1758f945a
76 schema:url http://www.nature.com/articles/1705895
77 sgo:license sg:explorer/license/
78 sgo:sdDataset articles
79 rdf:type schema:ScholarlyArticle
80 N0e4017affd9b4c43a54d4a8ed25b2cf1 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
81 schema:name Clinical Trials as Topic
82 rdf:type schema:DefinedTerm
83 N0eb3538e00014a0580d03910a6937d5d schema:name readcube_id
84 schema:value cfcba49d0c18951ef93c5a63cd783cdd63e4ac89bfe7864326addc8077a9b5d4
85 rdf:type schema:PropertyValue
86 N1617f63044a842b29810405e21342d93 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
87 schema:name Infant
88 rdf:type schema:DefinedTerm
89 N1682579dda9448cb9b69486feacf31b0 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
90 schema:name Child, Preschool
91 rdf:type schema:DefinedTerm
92 N19c161f3da774c3b969f81913b486967 schema:name nlm_unique_id
93 schema:value 8702459
94 rdf:type schema:PropertyValue
95 N1ccdfbe4f24b49ed96f6bb80b1467fe2 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
96 schema:name Transplantation, Homologous
97 rdf:type schema:DefinedTerm
98 N2ba5a3d3aae64d1a8949f293d421c99d schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
99 schema:name Genetic Therapy
100 rdf:type schema:DefinedTerm
101 N2c7b28fdcc1a44a3a44c551b9cc916e3 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
102 schema:name Humans
103 rdf:type schema:DefinedTerm
104 N362a3dc80c2344b198e18823a65c9b43 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
105 schema:name Immunologic Deficiency Syndromes
106 rdf:type schema:DefinedTerm
107 N38759fa5c1704c998542d81bf4be8ece schema:name dimensions_id
108 schema:value pub.1043623845
109 rdf:type schema:PropertyValue
110 N3ef04a951f914e88b9a07150b48bb419 schema:name doi
111 schema:value 10.1038/sj.bmt.1705895
112 rdf:type schema:PropertyValue
113 N4348c7e7374a4c9c9468fa56454490ad rdf:first sg:person.01231045607.82
114 rdf:rest rdf:nil
115 N46dccfae47e044f7a4c6ee85279901fb schema:volumeNumber 41
116 rdf:type schema:PublicationVolume
117 Nc1ca8cdfd793470aa0c6bbe4d132d2f4 schema:name pubmed_id
118 schema:value 17994122
119 rdf:type schema:PropertyValue
120 Nd8f11bf9781f424bb0cd42c81cbe93bb schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
121 schema:name Antigens, CD34
122 rdf:type schema:DefinedTerm
123 Nec86e8b553f949a6b73ee7a1758f945a schema:name Springer Nature - SN SciGraph project
124 rdf:type schema:Organization
125 Nf84f2bb9b53244638388f75d9c55d0db schema:issueNumber 2
126 rdf:type schema:PublicationIssue
127 Nfb5294aef777446085a729ab98c7a6cc schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
128 schema:name Hematopoietic Stem Cell Transplantation
129 rdf:type schema:DefinedTerm
130 anzsrc-for:10 schema:inDefinedTermSet anzsrc-for:
131 schema:name Technology
132 rdf:type schema:DefinedTerm
133 anzsrc-for:1004 schema:inDefinedTermSet anzsrc-for:
134 schema:name Medical Biotechnology
135 rdf:type schema:DefinedTerm
136 sg:journal.1096890 schema:issn 0268-3369
137 1476-5365
138 schema:name Bone Marrow Transplantation
139 rdf:type schema:Periodical
140 sg:person.01231045607.82 schema:affiliation https://www.grid.ac/institutes/grid.42505.36
141 schema:familyName Kohn
142 schema:givenName D B
143 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01231045607.82
144 rdf:type schema:Person
145 sg:pub.10.1038/35017565 schema:sameAs https://app.dimensions.ai/details/publication/pub.1021691346
146 https://doi.org/10.1038/35017565
147 rdf:type schema:CreativeWork
148 sg:pub.10.1038/nature03556 schema:sameAs https://app.dimensions.ai/details/publication/pub.1040117781
149 https://doi.org/10.1038/nature03556
150 rdf:type schema:CreativeWork
151 sg:pub.10.1038/nbt1216 schema:sameAs https://app.dimensions.ai/details/publication/pub.1053690685
152 https://doi.org/10.1038/nbt1216
153 rdf:type schema:CreativeWork
154 sg:pub.10.1038/nm0502-423 schema:sameAs https://app.dimensions.ai/details/publication/pub.1026599498
155 https://doi.org/10.1038/nm0502-423
156 rdf:type schema:CreativeWork
157 sg:pub.10.1038/nm0798-775 schema:sameAs https://app.dimensions.ai/details/publication/pub.1042866864
158 https://doi.org/10.1038/nm0798-775
159 rdf:type schema:CreativeWork
160 sg:pub.10.1038/nm0896-876 schema:sameAs https://app.dimensions.ai/details/publication/pub.1010628157
161 https://doi.org/10.1038/nm0896-876
162 rdf:type schema:CreativeWork
163 sg:pub.10.1038/nm1095-1017 schema:sameAs https://app.dimensions.ai/details/publication/pub.1013283766
164 https://doi.org/10.1038/nm1095-1017
165 rdf:type schema:CreativeWork
166 sg:pub.10.1038/nm1393 schema:sameAs https://app.dimensions.ai/details/publication/pub.1043834387
167 https://doi.org/10.1038/nm1393
168 rdf:type schema:CreativeWork
169 https://app.dimensions.ai/details/publication/pub.1082981291 schema:CreativeWork
170 https://app.dimensions.ai/details/publication/pub.1083034885 schema:CreativeWork
171 https://app.dimensions.ai/details/publication/pub.1083191032 schema:CreativeWork
172 https://app.dimensions.ai/details/publication/pub.1083254987 schema:CreativeWork
173 https://doi.org/10.1001/jama.295.5.508 schema:sameAs https://app.dimensions.ai/details/publication/pub.1016979749
174 rdf:type schema:CreativeWork
175 https://doi.org/10.1006/mthe.2000.0034 schema:sameAs https://app.dimensions.ai/details/publication/pub.1005421718
176 rdf:type schema:CreativeWork
177 https://doi.org/10.1016/j.clim.2005.07.006 schema:sameAs https://app.dimensions.ai/details/publication/pub.1013539255
178 rdf:type schema:CreativeWork
179 https://doi.org/10.1016/j.exphem.2006.05.023 schema:sameAs https://app.dimensions.ai/details/publication/pub.1049719462
180 rdf:type schema:CreativeWork
181 https://doi.org/10.1016/j.ymthe.2006.06.007 schema:sameAs https://app.dimensions.ai/details/publication/pub.1010867996
182 rdf:type schema:CreativeWork
183 https://doi.org/10.1016/s0065-2660(05)54009-4 schema:sameAs https://app.dimensions.ai/details/publication/pub.1016961895
184 rdf:type schema:CreativeWork
185 https://doi.org/10.1016/s0140-6736(03)12513-5 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012975233
186 rdf:type schema:CreativeWork
187 https://doi.org/10.1016/s0140-6736(04)17590-9 schema:sameAs https://app.dimensions.ai/details/publication/pub.1020712301
188 rdf:type schema:CreativeWork
189 https://doi.org/10.1016/s0140-6736(68)92673-1 schema:sameAs https://app.dimensions.ai/details/publication/pub.1007159919
190 rdf:type schema:CreativeWork
191 https://doi.org/10.1056/nejm199902183400703 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012177588
192 rdf:type schema:CreativeWork
193 https://doi.org/10.1056/nejmoa012616 schema:sameAs https://app.dimensions.ai/details/publication/pub.1044007615
194 rdf:type schema:CreativeWork
195 https://doi.org/10.1073/pnas.0608138103 schema:sameAs https://app.dimensions.ai/details/publication/pub.1011766231
196 rdf:type schema:CreativeWork
197 https://doi.org/10.1073/pnas.122131099 schema:sameAs https://app.dimensions.ai/details/publication/pub.1014781978
198 rdf:type schema:CreativeWork
199 https://doi.org/10.1089/hum.1990.1.3-257 schema:sameAs https://app.dimensions.ai/details/publication/pub.1059271001
200 rdf:type schema:CreativeWork
201 https://doi.org/10.1126/science.1070104 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062446366
202 rdf:type schema:CreativeWork
203 https://doi.org/10.1126/science.1083413 schema:sameAs https://app.dimensions.ai/details/publication/pub.1024101746
204 rdf:type schema:CreativeWork
205 https://doi.org/10.1126/science.1088547 schema:sameAs https://app.dimensions.ai/details/publication/pub.1018519577
206 rdf:type schema:CreativeWork
207 https://doi.org/10.1126/science.1091667 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062449108
208 rdf:type schema:CreativeWork
209 https://doi.org/10.1126/science.270.5235.470 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062551476
210 rdf:type schema:CreativeWork
211 https://doi.org/10.1126/science.270.5235.475 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062551477
212 rdf:type schema:CreativeWork
213 https://doi.org/10.1126/science.272.5259.263 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062552729
214 rdf:type schema:CreativeWork
215 https://doi.org/10.1126/science.288.5466.669 schema:sameAs https://app.dimensions.ai/details/publication/pub.1019730668
216 rdf:type schema:CreativeWork
217 https://doi.org/10.1128/mcb.21.12.3926-3934.2001 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012411175
218 rdf:type schema:CreativeWork
219 https://doi.org/10.1172/jci31659 schema:sameAs https://app.dimensions.ai/details/publication/pub.1042616999
220 rdf:type schema:CreativeWork
221 https://doi.org/10.1172/jci31661 schema:sameAs https://app.dimensions.ai/details/publication/pub.1035515942
222 rdf:type schema:CreativeWork
223 https://doi.org/10.1172/jci31666 schema:sameAs https://app.dimensions.ai/details/publication/pub.1023711644
224 rdf:type schema:CreativeWork
225 https://doi.org/10.1172/jci32949 schema:sameAs https://app.dimensions.ai/details/publication/pub.1028986816
226 rdf:type schema:CreativeWork
227 https://doi.org/10.1182/blood-2003-05-1558 schema:sameAs https://app.dimensions.ai/details/publication/pub.1013720305
228 rdf:type schema:CreativeWork
229 https://doi.org/10.1182/blood-2004-01-0045 schema:sameAs https://app.dimensions.ai/details/publication/pub.1051628326
230 rdf:type schema:CreativeWork
231 https://doi.org/10.1182/blood-2006-03-006908 schema:sameAs https://app.dimensions.ai/details/publication/pub.1022143644
232 rdf:type schema:CreativeWork
233 https://www.grid.ac/institutes/grid.42505.36 schema:alternateName University of Southern California
234 schema:name Department of Molecular Microbiology and Immunology, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
235 Division of Research Immunology/Bone Marrow Transplantation, Department of Pediatrics, Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
236 rdf:type schema:Organization
 




Preview window. Press ESC to close (or click here)


...