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Therapeutic Approaches for New Discoveries


The Faculty of the Li Weibo Institute for Rare Diseases Research are dedicated to developing therapeutic approaches for rare inherited diseases for which there are no cure. Towards this goal, they are developing state-of-the-art technologies in the fields of gene therapy and genome editing.

 

GENE THERAPY

GENOME EDITING

 

 

Gene Therapy


Gene therapy refers to the delivery of a nucleic acid, such as DNA or RNA, into a patient’s cells as a treatment for a disease. The approach uses viral vectors (typically adeno-associated virus (AAV) vectors) to deliver the nucleic acid into a patient. For diseases caused by a mutant protein, the AAV vector can introduce normal copies of the defective gene. Alternatively, if a disease is caused by having too much of a protein (for example, SOD1 in ALS, or HTT in Huntington’s disease) the AAV vector can express a synthetic microRNA targeting the disease-causing gene and turn it off.

 

Investigators Developing Gene Therapy Approaches

Kevin Donahue, MD

Kevin Donahue, MD

Professor of Medicine, Director, Electrophysiology Research

Website

Kate Fitzgerald, PhD

Kate Fitzgerald, PhD

Professor of Medicine, Director of the Program in Innate Immunity

Fitzgerald Lab

terence flotte md

Terence Flotte, MD

Professor of pediatrics, Celia and Isaac Haidak Professor of Medical Education

Flotte Lab

Guangping Gao, Ph.D.

Guangping Gao, PhD

Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of microbiology and physiological systems, Penelope Booth Rockwell Professor in biomedical Research

Gao Lab

Hemant Khanna, PhD

Hemant Khanna, PhD

Associate Professor, Department of Ophthalmology & Visual Sciences

Khanna Lab

Jeremy Luban, MD

Jeremy Luban, MD

Professor, program in molecular medicine and biochemistry

Luban Lab

 

Genome Editing


Genome editing aims to correct the genetic defect in the patient’s DNA (or genome) and thereby cure the disease. Genome editing technology is advancing at a breathtaking rate. In particular, programmable nucleases based on the CRISPR/Cas9 system are enabling basic and translational research for the development of new therapeutics.

Investigators Developing Genome Editing Tools

Neil Aronin MD

Neil Aronin MD

Professor of medicine, co-director of the Neurotherapeutics Institute

Aronin Lab

Erik Sontheimer, PhD

Erik Sontheimer, PhD

Professor, RNA Therapeutics Institute

Sontheimer Lab

Scot Wolfe, PhD

Scot Wolfe, PhD

Professor of molecular, cell and cancer biology

Wolfe Lab

Wen Xue, PhD

Wen Xue, PhD

Assistant professor of RNA therapeutics

Xue Lab
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Therapeutic Approaches for New Discoveries

 

Rare Diseases & Conditions

There are more than 7,000 diseases defined as “rare” by the National Institutes of Health, generally those that impact fewer than 200,000 people. Please select disease or condition from the list below for more information about the latest in research, clinical trials and therapy strategies. 

Rare Diseases

Education and Research Opportunities

Li Weibo Institute for Rare Diseases Research is home to the world-class preeminent researchers, who are committed for years to uncovering new discoveries. Working closely together in this uniquely collaborative environment, they seek to identify and develop solutions to devastating ailments, from heart disease to ALS. Their efforts have the potential to unlock therapies or even cures for people with a variety of rare diseases.