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 …

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo
gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer …

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

The RNA-guided endonuclease Cas9 has emerged as a versatile genome-editing platform.
However, the size of the commonly used Cas9 from Streptococcus pyogenes (SpCas9) …

Improved delivery of adeno-associated virus (AAV) vectors to the CNS will greatly enhance
their clinical utility. Selection of AAV9 variants in a mouse model led to the isolation of a …

Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas
(CRISPR-associated) genome editing systems have become one of the most robust …

Recombinant Adeno-associated virus vectors (rAAV) are widely used for gene delivery and
multiple naturally occurring serotypes have been harnessed to target cells in different …