Forbes July 31, 2024
A recent Harvard study introduces a new method for treating spinal muscular atrophy. The study used “base editing” to target the disease’s root cause by modifying the survival motor neuron gene. For many years, a rare condition called spinal muscular atrophy (SMA) has brought heartbreak to families around the world. This genetic disorder affects a baby’s ability to move, breathe, and grow normally. It has been the leading genetic cause of infant mortality, forcing parents to witness their young children battling this problematic condition, facing an unimaginable struggle.
Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants with this condition often struggle with basic activities like lifting their heads, rolling over,...
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