Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH.
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.
Sci Transl Med. 2011 Mar 2;3(72):72ra18.
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The work by Passini et al. reports the latest progress in development of antisense oligonucleotides (ASOs) as a therapy for spinal muscular atrophy (SMA). The report marks two important achievements in the aim to bring this potential therapeutic to the clinic. First, a single dose of ASOs can increase survival by more than 50 percent in a mouse model engineered to have a severe form of SMA. Second, the ASOs can be targeted to motor neurons in non-human primates.
This study is not only encouraging for the development of an ASO-based therapy for SMA, but also opens doors for exploring this therapeutic approach in the treatment of other diseases. An important next step will be to assess the ability of the ASOs to act as a long-term treatment of the disease in mouse models as well as larger animal models of the disease as they become available.