Adeno-associated virus vector-based gene therapy for hereditary diseases: current problems of application and approaches to solve them

Adeno-associated virus vector-based gene therapy for hereditary diseases: current problems of application and approaches to solve them

INTRODICTION. Currently, gene therapy based on adeno-associated virus (AAV) vectors faces a  number of barriers, both biomedical and technological, which require studying and overcoming for further development of this gene therapy technology.AIM. This study aimed to analyse the use of gene therapy f...

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Bibliographic Details
Main Authors: T. V. Egorova, A. A. Piskunov, D. A. Poteryaev
Format: Article
Language:Russian
Published: Ministry of Health of the Russian Federation. Federal State Budgetary Institution «Scientific Centre for Expert Evaluation of Medicinal Products» 2024-07-01
Series:Биопрепараты: Профилактика, диагностика, лечение
Subjects:
gene therapy
gene therapy product
adeno-associated virus
aav
adeno-associated virus vectors
hereditary diseases
haemophilia
duchenne muscular dystrophy
gene therapy efficacy
gene therapy safety
aav production technology
aav modification
triple-plasmid transfection
aav-particle purification
Online Access:https://www.biopreparations.ru/jour/article/view/572
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