Treatment

Lentiviral vector transduced CD34+ cells clinical trials

2 indexed studies · 2 currently recruiting

RecruitingPHASE1
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
This study is testing a new gene therapy for baby boys with a very rare inherited condition called SCID-X1, sometimes called 'bubble boy disease'. This therapy aims to correct the genetic fault that causes the condition, offering a new treatment option for those without a matched donor.
For: Severe Combined Immunodeficiency, X-Linked
United Kingdom
RecruitingPHASE1, PHASE2
Lentiviral Gene Therapy for p47 AR-CGD
This study looks at a new gene therapy for a rare immune condition called p47 AR-CGD, which causes severe infections and inflammation. It aims to correct the faulty gene by adding a healthy copy, helping the body fight off germs better.
For: P47-Phox, Deficiency of
United Kingdom