Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

This study is offering hematopoietic cell transplantation (HCT, also known as bone marrow or stem cell transplant) to patients with primary immune deficiencies, immune dysregulatory disorders, and inherited bone marrow failure syndromes — including rare conditions like Wiskott-Aldrich syndrome, SCID, Fanconi anemia, Diamond Blackfan anemia, and many others. For many of these patients, transplant is the only potential cure. The study uses a range of donor types (matched siblings, unrelated matched donors, and haploidentical family members) and carefully tailored conditioning regimens to optimize outcomes. By enrolling patients across three cohorts — primary immune deficiencies, short telomere syndromes, and Fanconi anemia/DNA repair disorders — researchers aim to continuously improve transplant protocols for these rare, life-threatening conditions. You may be eligible if: - You have a confirmed diagnosis of a primary immune deficiency, immune dysregulatory syndrome, or inherited bone marrow failure disorder with an indication for transplant - You have an available donor (matched sibling, matched unrelated, or haploidentical family member) - Your organs (heart, liver, kidneys, lungs) function adequately - Your Karnofsky or Lansky performance status is 70% or higher You may NOT be eligible if: - You have had a previous allogeneic stem cell transplant - You have an uncontrolled bacterial, viral, or fungal infection at enrollment - You are pregnant or breastfeeding - You are HIV-positive or have active hepatitis B or C - You have a diagnosis of idiopathic aplastic anemia Talk to your doctor to see if this trial is right for you.