CRISPR Targets NOTCH2NLC GGC Repeats to Treat NIID

CRISPR Technique Targets GGC Repeat Expansions in NOTCH2NLC Gene to Address Neuronal Intranuclear Inclusion Disease

Researchers have developed a CRISPR/Cas9-based techniqu […] The post CRISPR Technique Targets GGC Repeat Expansions in NOTCH2NLC Gene to Address Neuronal Intranuclear Inclusion Disease first appeared on GeneOnline News. The post CRISPR Technique Targets GGC Repeat Expansions in NOTCH2NLC Gene to Address Neuronal Intranuclear Inclusion Disease appeared first on GeneOnline News.

CRISPR Targets NOTCH2NLC GGC Repeats to Treat NIID

In an unprecedented leap forward for neurogenetics and therapeutic gene editing, researchers have pioneered a strikingly precise CRISPR/Cas9-based strategy to excise pathogenic nucleotide expansions within the NOTCH2NLC gene, heralding new hope for treating neuronal intranuclear inclusion disease (NIID). This debilitating neurodegenerative disorder, characterized by the accumulation of toxic nuclear inclusions and progressive neuronal loss, has …