Patients with the disease Spinal Muscular Atrophy have lost function of the SMN1 gene. The human genome also carries the

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Patients with the disease Spinal Muscular Atrophy have lost function of the SMN1 gene. The human genome also carries the

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Patients With The Disease Spinal Muscular Atrophy Have Lost Function Of The Smn1 Gene The Human Genome Also Carries The 1
Patients With The Disease Spinal Muscular Atrophy Have Lost Function Of The Smn1 Gene The Human Genome Also Carries The 1 (35.65 KiB) Viewed 179 times
Patients with the disease Spinal Muscular Atrophy have lost function of the SMN1 gene. The human genome also carries the SMN2 gene which potentially encodes an identical protein but whose mRNAs are spliced such that exon 6 is joined to exon 8, skipping exon 7. New drugs to treat Spinal Muscular Atrophy Earget this SMN2 exon 7 to stimulate its splicing. One of these drugs is an antisense oligonucleotide (ASO) that base pairs with the intron downstream of exon 7. What kind of sequence does the ASO target and how does this increase splicing of SMN2 exon 7?
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