Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
Adult
Antibodies, Neutralizing
/ immunology
Antibodies, Viral
/ immunology
Capsid
/ immunology
Cross Reactions
Dependovirus
/ genetics
Factor IX
/ metabolism
Follow-Up Studies
Gene Transfer Techniques
/ adverse effects
Genetic Therapy
/ adverse effects
Genetic Vectors
/ administration & dosage
Hemophilia B
/ therapy
Hepatocytes
/ metabolism
Humans
Infusions, Intra-Arterial
/ adverse effects
Liver
/ drug effects
Longitudinal Studies
Male
Middle Aged
Signal Transduction
/ drug effects
Treatment Outcome
Young Adult
AAV
adeno-associated virus
clinical gene therapy
gene therapy
hemophilia
neutralizing antibody
Journal
Molecular therapy : the journal of the American Society of Gene Therapy
ISSN: 1525-0024
Titre abrégé: Mol Ther
Pays: United States
ID NLM: 100890581
Informations de publication
Date de publication:
02 09 2020
02 09 2020
Historique:
received:
25
04
2020
revised:
26
05
2020
accepted:
03
06
2020
pubmed:
20
6
2020
medline:
28
8
2021
entrez:
20
6
2020
Statut:
ppublish
Résumé
Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durability, and the nature of the human immune response to AAV vectors. Here, we present longitudinal follow-up data of subjects who participated in the first trial of a systemically delivered AAV vector. Adult males (n = 7) with severe hemophilia B received an AAV2 vector at doses ranging from 8 × 10
Identifiants
pubmed: 32559433
pii: S1525-0016(20)30291-4
doi: 10.1016/j.ymthe.2020.06.001
pmc: PMC7474338
pii:
doi:
Substances chimiques
Antibodies, Neutralizing
0
Antibodies, Viral
0
Factor IX
9001-28-9
Types de publication
Clinical Trial
Journal Article
Multicenter Study
Observational Study
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
2073-2082Subventions
Organisme : NHLBI NIH HHS
ID : K08 HL140078
Pays : United States
Organisme : NHLBI NIH HHS
ID : K08 HL146991
Pays : United States
Organisme : NHLBI NIH HHS
ID : P01 HL064190
Pays : United States
Informations de copyright
Copyright © 2020. Published by Elsevier Inc.
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