Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.

Borges B, Brown SM, Chen WJ, Clarke MT, Herzeg A, Park JH, Ross J, Kong L, Denton M, Smith AK, Lum T, Zada FM, Cordero M, Gupta N, Cook SE, Murray H, Matson J, Klein S, Bennett CF, Krainer AR, MacKenzie TC, Sumner CJ. Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy. Sci Transl Med. 2025 May 14; 17(798):eadv4656.