Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial-Reference-Cited by-同舟云学术

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

Published:2011-05 Issue:5 Volume:22 Page:595-604
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Allay James A.¹,Sleep Susan¹,Long Scott¹,Tillman David M.¹,Clark Rob¹,Carney Gael²,Fagone Paolo²,McIntosh Jenny H.³,Nienhuis Arthur W.²,Davidoff Andrew M.⁴,Nathwani Amit C.³,Gray John T.²

Affiliation:

1. Children's GMP, Memphis, TN 38105.

2. Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105.

3. UCL Cancer Institute, University College London, London WC1E 6BT, United Kingdom.

4. Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN 38105.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2010.202

Reference52 articles.

1. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy

2. Identification and elimination of replication-competent adeno-associated virus (AAV) that can arise by nonhomologous recombination during AAV vector production

3. High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

4. Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

5. A Versatile and Scalable Two-Step Ion-Exchange Chromatography Process for the Purification of Recombinant Adeno-associated Virus Serotypes-2 and -5

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