Gene therapy for hemophilia: Current status and laboratory consequences.
Animals
Blood Coagulation
Blood Coagulation Tests
Clinical Trials as Topic
Dependovirus
/ genetics
Factor IX
/ genetics
Factor VIII
/ genetics
Genetic Therapy
/ adverse effects
Genetic Vectors
/ administration & dosage
Hemophilia A
/ blood
Hemophilia B
/ blood
Humans
Mutation
Phenotype
Treatment Outcome
FIX
FVIII
gene therapy
hemophilia
laboratory testing
Journal
International journal of laboratory hematology
ISSN: 1751-553X
Titre abrégé: Int J Lab Hematol
Pays: England
ID NLM: 101300213
Informations de publication
Date de publication:
Jul 2021
Jul 2021
Historique:
revised:
12
04
2021
received:
23
02
2021
accepted:
21
04
2021
entrez:
21
7
2021
pubmed:
22
7
2021
medline:
10
8
2021
Statut:
ppublish
Résumé
Since the cloning and characterization of the factor VIII (FVIII) and factor IX genes in the mid-1980s, gene therapy has been perceived as having significant potential for the treatment of severe hemophilia. Now, some 35 years later, these proposals are close to being realized through the licensing of the first clinical gene therapy product. Adeno-associated viral vector-mediated gene therapy for hemophilia A and B has been extensively investigated in preclinical models over the past 20 years, and since 2011, there has been increasing evidence in early phase clinical trials that this therapeutic strategy can provide safe and effective rescue of the hemostatic phenotype in severe hemophilia. As the uptake of hemophilia gene therapy progresses, it is clear that many aspects of the gene therapy process require crucial laboratory support to ensure safe and effective outcomes from his new therapeutic paradigm. These laboratory contributions extend from evaluations of the gene therapy vehicle, assessments of the patient immune status for the vector, and ultimately the performance of assays to determine the hemostatic benefit of the gene therapy and potentially of its long-term safety on the host genome. As with many aspects of past hemophilia care, the safe and effective delivery of gene therapy will require an informed and coordinated contribution from laboratory science.
Substances chimiques
F8 protein, human
839MOZ74GK
Factor VIII
9001-27-8
Factor IX
9001-28-9
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
117-123Subventions
Organisme : Canadian Institutes of Health Research (CIHR)
ID : FDN 154285
Informations de copyright
© 2021 John Wiley & Sons Ltd.
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