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 ...

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 ...

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 ...

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 ...

(L to R) First author Anoop Babu Vasandan, PhD, St. Jude Department of Immunology, co-corresponding author Caitlin Zebley, MD, PhD, St. Department of Bone Marrow Transplantation & Cellular Therapy and ...

DUBLIN – Bidding to become the second European firm to win a gene therapy approval, Glaxosmithkline plc filed a marketing authorization application (MAA) with the EMA for GSK2696273, a treatment for ...

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...