Which disease was among the first targets of gene therapy?
Adenosine deaminase deficiency autosomal recessive SCID became earliest successful gene therapy target due biological features favoring correction. Enzyme chromosome 20q13.12 deaminates adenosine to inosine and deoxyadenosine to deoxyinosine, deficiency leads accumulation deoxyadenosine converted deoxycytidine kinase to dATP elevated dATP allosterically inhibits ribonucleotide reductase essential dNTP synthesis DNA replication lymphocyte clonal expansion triggers apoptosis intrinsic pathway resulting absent T B NK cells. Lymphoid lineage provides selective advantage because corrected cells det
Ref: Lancet ADA First Gene Therapy NEJM 1990; NCBI SCID Gene Therapy History; NCERT Biotechnology Applications ADA Chapter 12.
