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Featured
Collaboration in Developing New Cell Therapy
In this video, Bob S. Carter, MD, PhD, Kwang-Soo Kim, PhD, Todd M. Herrington, MD, PhD, and Jeffrey S. Schweitzer, MD, PhD, discuss their cross-disciplinary and multi-institutional collaboration to develop this new therapy.
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Featured
Gene Therapy Reverses Degeneration of the Retinal Pigment Epithelium in Preclinical Models
Gene therapy with Nrf2—a master transcription factor—preserves the retinal pigment epithelium in mouse models of retinitis pigmentosa, David M. Wu, MD, PhD, of the Department of Ophthalmology, and colleagues observed.
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Featured
Enhanced Interferon Signaling May Improve CAR T-cell Immunotherapy for Solid Tumors
Marcela V. Maus, MD, PhD, Rebecca C. Larson, PhD, and colleagues found that loss of genes in the interferon-gamma receptor signaling pathway is a mechanism of resistance to chimeric antigen receptor (CAR) T-cell therapy, suggesting a way to improve CAR T-cell treatment design for solid tumors.
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CRISPR-Cas9 Genome Editing Treats Genetic Hearing Loss by Promoting Outer Hair Cell Survival
Yong Tao, PhD, Veronica Lamas, PhD, and Zheng-Yi Chen, DPhil, have extended their previous murine research to show CRISPR-associated protein 9 (Cas9)-based genome editing can treat hearing loss associated with monogenic mutations in outer hair cell genes and digenic mutations affecting both inner and outer hair cells.
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Perspective: Using Information on Somatic Mutations to Direct Management of Neurological Disorders
Kristopher T. Kahle, MD, PhD, and colleagues explain that somatic variations detected from neurosurgical biopsies or resection of fresh brain tissue are beginning to suggest which existing targeted medications could be used as monotherapies or adjuncts to surgery for non-oncologic neurological diseases.
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Developing a Gene Therapy for an Extremely Rare Cerebrovascular Disease
Massachusetts General Hospital physician-scientist Patricia Musolino, MD, PhD, was a key investigator in a clinical trial that led to the first FDA-approved gene therapy for a cerebral disease. Her research is focused on studying rare genetic disorders to develop approaches that may lead to gene therapy treatments.
Gene and Cell Therapy Contributors
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Bob S. Carter, MD, PhD
Chief, Neurosurgery Service
Recent Article
Co-transplantation of Regulatory T Cells Improves Cell Replacement Therapy for Parkinson's Disease
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Jeffrey S. Schweitzer, MD, PhD
Neurosurgeon, Department of Neurosurgery, Massachusetts General Hospital, Assistant Professor of Neurosurgery, Harvard Medical School
Recent Article
Essential Tremor Treatment Advancements Using MRI-guided Focused Ultrasound: Q&A With Jeffrey S. Schweitzer, MD, PhD
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Jeremy Slade Abramson, MD
Director, Jon and JoAnn Hagler Center for Lymphoma, Mass General Cancer Center, Associate Professor of Medicine, Harvard Medical School
Recent Article
Neutropenia-driven Approach to G-CSF Use May Be Feasible With CAR T-Cell Therapy
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Luk Vandenberghe, PhD
Associate Professor of Ophthalmology, Harvard Medical School, Grousbeck Family Chair in Gene Therapy and Director of the Grousbeck Gene Therapy Center, Mass Eye and Ear
Recent Article
Developing a Gene-based Vaccine Candidate for COVID-19
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Marcela V. Maus, MD, PhD
Director, Cellular Immunotherapy Program, Mass General Cancer Center, Associate Professor of Medicine, Harvard Medical School
Recent Article
Novel Tandem CAR T-Cell Effective Against Heterogeneous Glioblastoma
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Merit Ester Cudkowicz, MD, MSc
Chief, Neurology Service, Director of the Sean M. Healey & AMG Center for ALS at Mass General, Director of Neurological Clinical Research Institute and the Jilieane Dorn Professor of Neurology, Harvard Medical School
Recent Article
ALS Therapy Research and Development: Insights from Merit Cudkowicz, MD, MSc
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Priscilla K. Brastianos, MD
Director, Central Nervous System Metastasis Center, Mass General Cancer Center, Associate Professor of Medicine, Harvard Medical School
Recent Article
Targeted Therapy for BRAF Mutation in Craniopharyngioma
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Robert (Mark) Richardson, MD, PhD
Director of Functional Neurosurgery, Massachusetts General Hospital
Recent Article
Intracranial Stereo-EEG Resolves Discordant Clinical vs. Neuroimaging Findings in Patient With Tonic–Clonic Seizures
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Todd M. Herrington, MD, PhD
Director, Deep Brain Stimulation Program, Massachusetts General Hospital
Recent Article
Co-transplantation of Regulatory T Cells Improves Cell Replacement Therapy for Parkinson's Disease
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William T. Curry, MD
Co-Director, Mass General Neuroscience , Director of Neurosurgical Oncology, Massachusetts General Hospital, Adeline Rose Wydotis Professor of Neurosurgery, Harvard Medical School
Recent Article
Review: The Role of Antibody-Based Therapies in Neuro-Oncology
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Yakeel T. Quiroz, PhD
Associate Professor, Harvard Medical School, Director, Familial Dementia Neuroimaging Lab, Director, Multicultural Alzheimer's Prevention Program, Massachusetts General Hospital, Paul B. and Sandra M. Edgerley MGH Research Scholar 2020-2025
Recent Article
Higher Levels of Education Buffer Against Autosomal Dominant Alzheimer's Disease
