
Patricia Musolino
Director Neurovascular Genetics and Gene Therapy Program · Mass General Brigham
Credentials: · · · · ·
History
- Present
Pediatric Stroke and Cerebrovascular Service · Mass General Brigham
Research
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
New England Journal of Medicine · 2017 · 455 citations
JAMA Neurology · 2020 · 132 citations
Brain endothelial dysfunction in cerebral adrenoleukodystrophy
Brain · 2015 · First of 10 authors · 72 citations

14 papers

10 papers

7 papers

6 papers

5 papers
- SL
5 papers

4 papers
- OR
4 papers
Grants
- 2026–2031
In vivo Genome Editing for Cerebrovascular Disorders
National Institute of Neurological Disorders and Stroke · R01
PI · $616.1K · Massachusetts General Hospital
- 2026–2029
National Institute of Neurological Disorders and Stroke · U01
Contact PI · $2.5M · Massachusetts General Hospital
- 2023–2028
Gene Therapy for Disorders of the Extracellular Matrix
National Institute of Neurological Disorders and Stroke · R01
PI · $822K · University of California, San Francisco
- 2022–2027
Modeling and Therapeutic Approaches for Genetic Vasculopathies
National Institute of Neurological Disorders and Stroke · R01
Contact PI · $3.5M · Massachusetts General Hospital
Clinical trials
- 2023–2028
Mucolipidosis Type IV Natural History Study
Principal investigator · Active not recruiting
Massachusetts General Hospital


