Genome and Gene Therapy

Genome-editing Technologies for Gene and Cell Therapy.

Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the...


Genome Surgery and Gene Therapy in Retinal Disorders

The emergence of genome surgery techniques like the clustered regularly interspaced short palindromic repeats (CRISPR) editing technology has given researchers a powerful tool for precisely introducing targeted changes within the genome. New modifications to the CRISPR-Cas system have been made since its recent discovery, such as high-fidelity Cas9 variants to reduce off-target effects and tran...

ژن درمانی Gene Therapy

Genome Editing Gene Therapy for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by loss of function of the dystrophin gene on the X chromosome. Gene augmentation of dystrophin is challenging due to the large size of the dystrophin cDNA. Emerging genome editing technologies, such as TALEN and CRISPR-Cas9 systems, open a new erain the restoration of functional dystrophin and are a hallmark of bona fide gen...

Gene and genome duplication.

Genomic sequencing projects have revealed the productivity of processes duplicating genes or entire chromosome segments. Substantial proportions of the yeast, Arabidopsis and human gene complements are made up of duplicates. This has prompted much interest in the processes of duplication, functional divergence and loss of genes, has renewed the debate on whether an early vertebrate genome was t...