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 …

Clustered regularly interspaced short palindromic repeats (CRISPR) system provides
adaptive immunity against plasmids and phages in prokaryotes. This system inspires the …

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or
treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced …

Adeno-associated virus (AAV) vectors have emerged as a gene-delivery platform with
demonstrated safety and efficacy in a handful of clinical trials for monogenic disorders …

Adeno-associated virus (AAV) serotypes differ broadly in transduction efficacies and tissue
tropisms and thus hold enormous potential as vectors for human gene therapy. In reality …

Adeno-associated viral vectors are highly safe and efficient gene delivery vehicles.
However, numerous challenges in vector design remain, including neutralizing antibody …

▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic
material into a cell, tissue, or whole organ, with the goal of curing a disease or at least …

Adeno-associated virus (AAV) has shown promising therapeutic efficacy with a good safety
profile in a wide range of animal models and human clinical trials. With the advent of …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …