TY  - JOUR
AU  - Mulcahy, Pádraig J.
AU  - Iremonger, Kayleigh
AU  - Karyka, Evangelia
AU  - Herranz Martín, Saúl
AU  - Shum, Ka-To
AU  - Van Tam, Janice Kal
AU  - Azzouz, Mimoun
PY  - 2014
DO  - 10.1089/hum.2013.186
SN  - 1043-0342
SN  - 1557-7422
UR  - https://hdl.handle.net/20.500.14352/96282
T2  - Human Gene Therapy
AB  - Spinal muscular atrophy (SMA) is a severe autosomal recessive disease caused by a genetic defect in the survival motor neuron 1 (SMN1) gene, which encodes SMN, a protein widely expressed in all eukaryotic cells. Depletion of the SMN protein causes...
LA  - eng
PB  - Mary Ann Liebert
TI  - Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy
TY  - journal article
VL  - 25
ER  -