메뉴 건너뛰기




Volumn 5, Issue 1, 2013, Pages 1-3

Gene therapy enters the pharma market: The short story of a long journey

Author keywords

[No Author keywords available]

Indexed keywords

ALIPOGENE TIPARVOVEC; CYCLOSPORIN A; LIPOPROTEIN LIPASE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER;

EID: 84871991715     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.1002/emmm.201202291     Document Type: Note
Times cited : (78)

References (14)
  • 2
    • 84871269515 scopus 로고    scopus 로고
    • Familial lipoprotein lipase deficiency.
    • Pagon RA, Bird TD, Dolan CR, Stephens K, Adam MP (eds) Seattle (WA): University of Washington
    • Brunzell JD (1999) Familial lipoprotein lipase deficiency. In GeneReviews™ [Internet], Pagon RA, Bird TD, Dolan CR, Stephens K, Adam MP (eds) Seattle (WA): University of Washington
    • (1999) GeneReviews™ [Internet]
    • Brunzell, J.D.1
  • 6
    • 84875962928 scopus 로고    scopus 로고
    • Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy for lipoprotein lipase deficiency: an open-label trial
    • DOI: 10.1038/gt.2012.43
    • Gaudet D, Methot J, Dery S, Brisson D, Essiembre C, Tremblay G, Tremblay K, de Wal J, Twisk J, van den Bulk N et al (2012a) Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther DOI: 10.1038/gt.2012.43
    • (2012) Gene Ther
    • Gaudet, D.1    Methot, J.2    Dery, S.3    Brisson, D.4    Essiembre, C.5    Tremblay, G.6    Tremblay, K.7    de Wal, J.8    Twisk, J.9    van den Bulk, N.10
  • 7
    • 84863984123 scopus 로고    scopus 로고
    • Gene therapy for lipoprotein lipase deficiency
    • Gaudet D, Methot J, Kastelein J (2012b) Gene therapy for lipoprotein lipase deficiency. Curr Opin Lipidol 23: 310- 320
    • (2012) Curr Opin Lipidol , vol.23 , pp. 310-320
    • Gaudet, D.1    Methot, J.2    Kastelein, J.3
  • 9
    • 79954650563 scopus 로고    scopus 로고
    • State-of-the-art gene-based therapies: the road ahead
    • Kay MA (2011) State-of-the-art gene-based therapies: the road ahead. Nat Rev Genet 12: 316- 328
    • (2011) Nat Rev Genet , vol.12 , pp. 316-328
    • Kay, M.A.1
  • 10
    • 79954622209 scopus 로고    scopus 로고
    • Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
    • Mingozzi F, High KA (2011) Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 12: 341- 355
    • (2011) Nat Rev Genet , vol.12 , pp. 341-355
    • Mingozzi, F.1    High, K.A.2
  • 14
    • 84866505684 scopus 로고    scopus 로고
    • Gene therapy to be authorised for first time in EU
    • Watts G (2012) Gene therapy to be authorised for first time in EU. Br Med J 345: e5009
    • (2012) Br Med J , vol.345
    • Watts, G.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.