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 ...
Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (ADA-SCID) is an ultrarare genetic disease that results in the accumulation of toxic adenine metabolites that ...
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