The Cold Spring Harbor Laboratory scientists have come to an interesting conclusion that spinal muscular atrophy (SMA) might not exclusively affect the motor neurons in the spinal cord as has long been thought. The new findings suggest that defects in peripheral tissues such as liver, muscle, heart, etc., might also contribute to the pathology of the disease in severely affected patients. The study, which also paves the way for the potential SMA drug ISIS SMNrx to enter human trials by the end of the year, appears in Nature on October 6.
The insight comes from testing an antisense therapy on mice with a severe form of SMA. The candidate drug ISIS SMNrx dramatically suppressed symptoms when simply injected under the animals’ skin. “These systemic, or subcutaneous, injections, extended the lifespan of mice that have the equivalent of severe human SMA by 25-fold,” reports CSHL’s Professor Adrian Krainer, Ph.D., who led the CSHL team in collaboration with a group led by Dr. Frank Bennett of California-based Isis Pharmaceuticals.
“However, we have yet to determine whether these findings are unique to this animal model of severe SMA—and by extension, relevant only to the patients with the most severe disease —or if they will be valid in other SMA types that manifest with milder, less severe symptoms,” cautions Dr. Bennett.
The antisense therapy uses anti-sense oligonucleotides (ASOs) —small, chemically modified bits of RNA that can be custom-designed to redirect splicing— to boost SMN levels by correcting SMN2 splicing.
Detailed information on CSHL website.
The insight comes from testing an antisense therapy on mice with a severe form of SMA. The candidate drug ISIS SMNrx dramatically suppressed symptoms when simply injected under the animals’ skin. “These systemic, or subcutaneous, injections, extended the lifespan of mice that have the equivalent of severe human SMA by 25-fold,” reports CSHL’s Professor Adrian Krainer, Ph.D., who led the CSHL team in collaboration with a group led by Dr. Frank Bennett of California-based Isis Pharmaceuticals.
“However, we have yet to determine whether these findings are unique to this animal model of severe SMA—and by extension, relevant only to the patients with the most severe disease —or if they will be valid in other SMA types that manifest with milder, less severe symptoms,” cautions Dr. Bennett.
The antisense therapy uses anti-sense oligonucleotides (ASOs) —small, chemically modified bits of RNA that can be custom-designed to redirect splicing— to boost SMN levels by correcting SMN2 splicing.
Detailed information on CSHL website.
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