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Antisense oligonucleotide therapy for CHD2 deficiency
- Development stage
- Unknown
- Lead developer
- Mahzi Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Antisense oligonucleotide (ASO) therapy for CHD2 deficiency is a precision medicine approach being developed to treat disorders caused by mutations in the CHD2 gene. CHD2-related disorders typically present with refractory epilepsy, global developmental delays, speech and motor delays, intellectual disability, behavioral problems, autism, photosensitivity, and regression. The therapy aims to modulate CHD2 expression through antisense oligonucleotides, which are single-stranded oligonucleotides designed to specifically target RNA of interest through Watson-Crick base pairing. This results in RNase H1-mediated mRNA degradation, modulation of pre-mRNA splicing, or regulation of translational efficiency. The approach is being developed in collaboration between Mahzi Therapeutics and the Ulitsky lab at the Weizmann Institute of Science.
- Other names
- ASO therapy for CHD2 deficiency
02
Targets
CHD2 (Down syndrome cell adhesion molecule)
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