Pipeline
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Current Nucleus Projects
Project | Collaborator | Discovery | Lead Identification | Lead Optimization | Preclinical Development |
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| Engineering AAV-CRISPR-TO as a Spatial RNA Therapeutic for ALS: A Dual-Vector Optimization Strategy | Stanley Qi |
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| Mapping cytokine signaling requirements for in vivo AAV-engineered T cells | Anusha Kalbasi |
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Current IMA Projects
Project | Collaborator | Lead Identification | Lead Optimization | Preclinical Development |
|---|---|---|---|---|
| AAV gene therapy for IPEX syndrome | Rosa Bacchetta |
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| AAV gene therapy for chronic pain | Xiaoke Chen |
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| Gene editing for HIV | Matthew Porteus |
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| CRISPR-corrected esophageal cell therapy for epidermolysis bullosa | Anthony Oro |
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| Tri-specific CAR T-cell therapy for medulloblastoma | Crystal Mackall |
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| Cell therapy for breast cancer | Livnat Jerby |
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Completed Projects
- AAV gene therapy for dilated cardiomyopathy (PI: Ioannis Karakikes)
- AAV gene therapy for glaucoma (PI: Yang Hu)
- Gene therapy for IL10 deficiency (PI: Maria Grazia Roncarolo)
- Guided knock-in and efficient non-viral CAR T-cell manufacturing (PI: Crystal Mackall)
- Engineering novel AAV for hemophilia gene therapy (PI: John Fraser Wright)