Adeno-associated virus as a delivery vector for gene therapy ...
2024年4月3日 · Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different...
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
2017年7月1日 · Adeno-associated virus (AAV) is a versatile viral vector technology that can be engineered for very specific functionality in gene therapy applications. To date, AAV has been shown to be safe and effective in preclinical and clinical settings.
Adeno-associated virus vector as a platform for gene therapy ...
2019年2月1日 · Adeno-associated virus (AAV) vectors represent the leading platform for therapeutic gene delivery, with two recombinant AAV gene therapy products having gained regulatory approval in Europe...
Adeno Associated Virus (AAV) for Cell and Gene Therapy
Adeno-associated virus (AAV) – based delivery techniques may hold the key. In the simplest form, translating gene therapies from conceptual design to clinical trials involve identifying a therapeutic gene, finding a means to deliver it and identifying a suitable route of administration.
Gene Therapy Using Adeno-Associated Virus Vectors
The lack of pathogenicity of the virus, the persistence of the virus, and the many available serotypes have increased AAV's potential as a delivery vehicle for gene therapy applications. This review will focus on the biology of AAV and its use as a vector for gene therapy.
Adeno-associated virus vector as a platform for gene therapy ...
Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States.
Adeno-Associated Virus at 50: A Golden Anniversary of ...
2015年5月1日 · The authors highlighted areas that required additional understanding, including expression from integrated AAV genomes, improved rescue of AAV, studies of larger DNA inserts, host range mutants, and gene therapy applications, all of which would assume over the …