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% ...

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), sometimes called "bubble boy disease," are born with a defective gene that prevents them from producing immune cells. Gene ...

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 ...

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 ...

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 ...

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 ...