AAV vectors: The Rubik's cube of human gene therapy
A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Fantastic AAV gene therapy vectors and how to find them—random diversification, rational design and machine learning
Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …
variety of species, tissues and cell types. For over half a century, their intriguing biology and …
[HTML][HTML] Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
Replacing or editing disease-causing mutations holds great promise for treating many
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …
Cone-shaped HIV-1 capsids are transported through intact nuclear pores
Summary Human immunodeficiency virus (HIV-1) remains a major health threat. Viral capsid
uncoating and nuclear import of the viral genome are critical for productive infection. The …
uncoating and nuclear import of the viral genome are critical for productive infection. The …
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
J El Andari, E Renaud-Gabardos, W Tulalamba… - Science …, 2022 - science.org
Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants
J Weinmann, S Weis, J Sippel, W Tulalamba… - Nature …, 2020 - nature.com
Adeno-associated virus (AAV) forms the basis for several commercial gene therapy products
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …
Production, processing, and characterization of synthetic AAV gene therapy vectors
J El Andari, D Grimm - Biotechnology journal, 2021 - Wiley Online Library
Over the last two decades, gene therapy vectors based on wild‐type Adeno‐associated
viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into …
viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into …
AAV capsid design: A Goldilocks challenge
In vivo therapeutic gene transfer has emerged as a novel class of medicines. Its feasibility
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …
[HTML][HTML] Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS
Background Adeno-associated virus (AAV) vectors are a promising vehicle for noninvasive
gene delivery to the central nervous system via intravenous infusion. However, naturally …
gene delivery to the central nervous system via intravenous infusion. However, naturally …
Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing
The CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 genome
editing system has been a major technological breakthrough that has brought revolutionary …
editing system has been a major technological breakthrough that has brought revolutionary …