Chemically Modified Oligonucleotides Silence Mutant SPTLC1 in an in vitro Model of HSAN1
Hereditary sensory and autonomic neuropathy type 1 (HSAN1) is a monogenic, autosomal dominantly inherited, neurodegenerative disorder resulting in loss of pain and temperature sensation in the distal limbs. HSAN1 is caused by point mutations in a single allele of serine palmitoyltransferase long cha...
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| Format: | Others |
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eScholarship@UMMS
2018
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| Online Access: | https://escholarship.umassmed.edu/gsbs_diss/994 https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=2001&context=gsbs_diss |