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You searched for: Author/Creator Broccoli, Vania

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1. Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery. (21st December 2017)

2. Coenzyme A corrects pathological defects in human neurons of PANK2‐associated neurodegeneration. Issue 10 (11th August 2016)

4. Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy. (16th September 2016)

5. Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy. (16th September 2016)

7. I10 A genome-wide screening in pluripotent cells identifies MTF1 as a novel suppressor of mutant huntingtin toxicity. (12th September 2022)

8. MSEL‐1L deficiency affects vasculogenesis and neural stem cell lineage commitment. Issue 4 (12th September 2017)

10. Rapid Generation of Functional Dopaminergic Neurons From Human Induced Pluripotent Stem Cells Through a Single‐Step Procedure Using Cell Lineage Transcription Factors. (8th May 2013)