Drugs / AAVrh.10CUCLN2
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT01414985 | Apr 2010 → Nov 2015 | late infantile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder | Weill Medical College of Cornell University | Completed | No outcome recorded |
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAVrh.10CUCLN2 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01414985 ↗ |
| Action | Restore | “delivering a good copy of the mutated gene to the nerve cells via a virus.” NCT01414985 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-associated Virus Gene Transfer Vector Serotype rh.10” NCT01414985 ↗ |