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Autor:
Flotte TR; Editor-in-Chief.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2020 May; Vol. 31 (9-10), pp. 489-490.
Autor:
Terence R. Flotte
Publikováno v:
Human gene therapy. 31(9-10)
Autor:
Barrie J. Carter
Publikováno v:
Viral Genome Methods ISBN: 9781003420163
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::718a67c03d872b6f76af8e43fc1ca914
https://doi.org/10.1201/9781003420163-15
https://doi.org/10.1201/9781003420163-15
Autor:
Barrie J. Carter
Publikováno v:
Human Gene Therapy. 33:577-578
My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective
Autor:
Barrie J. Carter
Publikováno v:
Human Gene Therapy
Autor:
Barrie J. Carter
Publikováno v:
Gene Therapy and Regulation. :5-30
Impressive progress has been made in clinical development of adeno-associated virus (AAV) vectors over the last 15 years in more than 40 clinical trials, involving many hundreds of subjects with vector delivery by many different routes and at a wide
Autor:
Barrie J. Carter, Beverly L. Davidson, Jodi L. McBride, Ryan L. Boudreau, Yi Xing, Inês Martins, Shihao Shen
Publikováno v:
Molecular Therapy. 17:1053-1063
Huntington's disease (HD) is a fatal neurodegenerative disease caused by mutant huntingtin (htt) protein, and there are currently no effective treatments. Recently, we and others demonstrated that silencing mutant htt via RNA interference (RNAi) prov
Publikováno v:
Clinical Virology. :351-370
Gene therapy is an emerging medical approach which seeks to apply molecular techniques to attack diseases at the fundamental level of the genes. The first techniques which were developed for genetic manipulation of mammalian cells involved direct int
Autor:
Alex Mas Monteys, Haim Burstein, Brian L. Gilmore, Barry A. Polisky, Ryan L. Boudreau, Jodi L. McBride, Barrie J. Carter, Richard W. Peluso, Beverly L. Davidson, Scott Q. Harper, Inâs Martins, Patrick D. Staber
Publikováno v:
Proceedings of the National Academy of Sciences. 105:5868-5873
Huntington's disease (HD) is a fatal, dominant neurodegenerative disease caused by a polyglutamine repeat expansion in exon 1 of the HD gene, which encodes the huntingtin protein. We and others have shown that RNAi is a candidate therapy for HD becau