The FDA approved Beremagene Geperpavec (B-VEC), also known by its trade name Vyjuvke, on May 19, 2023, for the treatment of dystrophic maculopapularity in patients aged 6 months and older with epidermolysisRead More…
A New Study Uncovers How RNA Editing Directs Immune Cells to Sites of Tissue Damage
In a recent study, researchers from the University of Heidelberg, Mannheim Medical School, and Newcastle University uncovered a novel mechanism of immune cell trafficking through the investigation of vascular disease. This knowledge mayRead More…
Gene-Edited Pigs May Avoid Viral Infections in Xenografts
Technological advances in organ transplantation have given hope to many patients with organ failure. However, the scarcity of organ donors remains a significant problem, resulting in many patients waiting for organ transplantsRead More…
Activating Oligonucleotide Technology as A Therapeutic Approach in Cancer
Cell growth is strictly regulated by tumor suppressor genes (TSGs), so that cells divide only when absolutely necessary and then respond to appropriate external signals, such as growth factors. In addition toRead More…
A Novel Gene Therapy May Effectively Delay Vision Loss in Bardet-Biedl Syndrome
Bardet-Biedl syndrome (BBS) is a classic ciliopathy. In this disease, mutations in genes essential for maintaining cilia function lead to cellular dysfunction in a variety of cell types, resulting in obesity, polydactyly,Read More…
AAV Delivery of Micro DMD Gene for the Treatment of Duchenne Muscular Dystrophy in GRMD Model
Duchenne muscular dystrophy (DMD) is a monogenic disease with a relatively high prevalence, caused by mutations in the gene encoding the anti-myotonic protein (Dystrophin) on the X chromosome, resulting in the failureRead More…
Novel Gene Therapy Holds Promise for Treating Children with Artemis-SCID
In a recent clinical study, researchers from the University of California, San Francisco, have pioneered a novel gene therapy that holds the promise of a healthier life for 10 young children whoRead More…
Novel Gene Therapy Targeting Overactive Brain Cells Holds Promise for Treating Neurological Disorders
In a recent study, researchers from University College London have developed a new approach to treating neurological and psychiatric disorders that works by reducing the neuronal firing of overactive brain cells. TheRead More…
AAV Gene Therapy Helps Restore Night Vision in Congenital Melanoma Patients Blind for Decades
The human brain mainly receives external information through five senses: sight, hearing, smell, touch, and taste, of which at least 80% of external information is obtained through vision. The loss of visionRead More…
Application Highlights and Future Development Trends of Gene Editing Technologies
What is Gene Editing? Gene editing is an emerging genetic engineering technology that can precisely modify specific target genes in an organism’s genome. Simply put, gene editing is the use of aRead More…