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 …

The CRISPR-Cas (clustered regularly interspaced short palindromic repeats-associated
proteins) technology enables rapid and precise genome editing at any desired genomic …

Recently, there have been revolutions in the development of both gene medicine therapy
and genome surgical treatments for inherited disorders. Much of this progress has been …

US9938521B2 - CRISPR/CAS-related methods and compositions for treating leber's
congenital amaurosis 10 (LCA10) - Google Patents US9938521B2 - CRISPR/CAS-related …

Gene augmentation and genome editing are promising strategies for the treatment of
monogenic inherited retinal diseases. Although gene augmentation treatments are …

The advent of gene editing has introduced the ability to make changes to the genome of
cells, thus allowing for correction of genetic mutations in patients with monogenic diseases …

In recent years, there has been accelerated growth of clustered regularly interspaced short
palindromic repeats (CRISPR) genome surgery techniques. Genome surgery holds promise …

Introduction: Genetic alterations resulting in a dysfunctional retinal pigment epithelium
and/or degenerating photoreceptors cause impaired vision. These juxtaposed cells in the …

Improvements in the understanding of human genetics and its roles in disease development
and prevention have led to an increased interest in therapeutic genome editing via the use …

Background: Despite the proof of principle that gene therapy can cure various monogenic
diseases, limited clinical progress has been noted for gene therapy of the respiratory …