Cell therapies using HSPCs have been approved under the commercial names of Libmeldy®, Skysona®, Strimvelis®, Zolgensma® and Zynteglo®, for the treatment of metachromatic leukodystrophy, early cerebral adrenoleukodystrophy, adenosine deaminase severe combined immune deficiency and beta thalassemia, respectively. This evidence concerns the gene ADA and Beta-thalassemia.