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DNAI1 gene therapy
- Development stage
- Preclinical
- Lead developer
- ReCode Therapeutics
- Modality
- Gene Therapies
- Administration
- Inhalation
01
Overview
DNAI1 gene therapy is an experimental gene addition approach designed to treat Primary Ciliary Dyskinesia (PCD) caused by biallelic mutations in the *DNAI1* gene. PCD is a rare genetic disorder characterized by defective motile cilia, which leads to impaired mucociliary clearance and chronic sino-oto-pulmonary infections. This therapy utilizes viral vectors, specifically lentivirus (LV) and adeno-associated virus (AAV), to deliver a functional copy of the porcine or human *DNAI1* cDNA to airway epithelial cells. By restoring the expression of the Dynein Axonemal Intermediate Chain 1 protein, the therapy aims to re-establish the assembly of ciliary outer dynein arms, thereby restoring ciliary beat frequency and mucociliary transport. Research is currently being conducted at the University of Iowa using a porcine DNAI1 knockout model that recapitulates the clinical hallmarks of the human disease.
- Other names
- DNAI1 gene addition therapyDNAI-1 gene addition therapyDNAI 1 gene addition therapy
02
Targets
DNAI1 (Dynein axonemal intermediate chain 1)
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