Gene therapy is superior to haploidentical hematopoietic stem cell transplantation (HSCT) to treat X-linked severe combined immunodeficiency (SCID-X1), according to a report published online April 13 ...

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...

X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in T-cell, B-cell, and natural killer (NK) cell function, caused by mutations in ...

Current GT strategies employ viral vectors (eg, murine g-retroviruses or lentiviruses) to introduce genetic material into isolated HSCs. However, gene editing (eg, CRISPR-based GT) is under active ...

Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the closing of a $4.6 million seed financing led by ...

Researchers headed by teams at Newcastle University, the Wellcome Sanger Institute, and the Great North Children’s Hospital have identified a single gene mutation common to 11 pediatric patients with ...

A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...

TUBA CITY, Ariz. - Hataałii Tiisyatonii "HT" Begay, a Native American boy who is Diné, or Navajo, rolls toy trucks around in the dirt. He chases chickens into their coop. He pauses to watch his dad ...

Nearly 30 US gene therapy trials were halted on Tuesday following the announcement that a second child in a pioneering French gene therapy trial has developed leukaemia following the treatment. The ...