Gene Therapy, Antisense Show Big Gains in Spinal Muscular Atrophy – Medscape
Medscape |
Gene Therapy, Antisense Show Big Gains in Spinal Muscular Atrophy
Medscape The two treatments — an antisense oligonucleotide drug and gene therapy — each enabled motor neurons of severely affected young children to produce a critical protein and induce clinical improvements. The effects of the gene therapy are particularly … |
