Видео 21
Просмотров 998

21:48

Meet the Researcher: Fiona Serack

3:50

Meet the Consortium: Ryan Spellecy

13:40

Meet the Researcher: Manda Arbab

6:27

Meet the Researcher: Shengdar Tsai

7:27

Meet the Researcher: David Spencer

4:27

Meet the Expert: Steve Gray and Kiran Musunuru

In this video of the Meet the Expert Series, Drs. Steve Gray and Kiran Musunuru share their experience and tips for preparing an IND application for gene therapy.
Steve Gray, PhD, is a Professor in the Department of Pediatrics at the University of Texas Southwestern (UTSW) Medical Center, as well as the co-director of the UTSW Gene Therapy Program and director of the UTSW Viral Vector Facility.
Kiran Musunuru, MD, PhD, MPH, ML, is a Professor of Medicine, the Director of the Genetic and Epigenetic Origins of Disease Program, and the Scientific Director at the Center for Inherited Cardiovascular Disease at Perelman School of Medicine (University of Pennsylvania).
Meet the Expert is a webina...

Видео

21:48

Meet the Consortium: Arthur Derse

Просмотров 16Месяц назад

Arthur Derse, MD, JD, FACEP is the Director of the Center for Bioethics and Medical Humanities and a Professor of Bioethics and Emergency Medicine at the Medical College of Wisconsin. He is part of the SCGE TCDC, where he provides insight on ELSI issues.

3:50

Meet the Researcher: Fiona Serack

Просмотров 23Месяц назад

Fiona Serack, PhD, is a Research Scientist at The Broad Institute. She is part of the SCGE project "Therapeutic Editing to Lower PrP in Prion Disease" led by Dr. Sonia Vallabh. Learn more at scge.mcw.edu/phase-2-ind-enabling-studies/#vallabh

13:40

Meet the Consortium: Ryan Spellecy

Просмотров 8Месяц назад

Ryan Spellecy, PhD is a Professor of Bioethics and the Assistant Provost and Director for the Medical College of Wisconsin's Human Research Protection Program. He is part of the SCGE TCDC, where he provides insight on ELSI issues. Learn more at scge.mcw.edu/phase-2-tcdc/

6:27

Meet the Researcher: Manda Arbab

Просмотров 13Месяц назад

Manda Arbab, PhD, is an Assistant Professor at Boston Children’s Hospital and Harvard Medical School. She is a PI on the SCGE phase 2 project titled "Preclinical Genome Editing for Rare Neurological Diseases". Here she discusses her SCGE work on Spinal Muscular Atrophy (SMA). Learn more at: scge.mcw.edu/phase-2-ind-enabling-studies/#jackson

7:27

Meet the Researcher: Shengdar Tsai

Просмотров 272 месяца назад

Shengdar Tsai, PhD is an Associate Member in the Department of Hematology at St. Jude Children's Research Hospital. He is the PI on two SCGE phase 2 projects: "Optimization and qualification of biochemical CHANGE-seq unbiased genome-wide activity and gRNA sequencing methods for therapeutic genome editing INDs" and "Optimization and qualification of cellular unbiased GUIDE-seq-2 genome-wide acti...

4:27

Meet the Researcher: David Spencer

Просмотров 32 месяца назад

David Spencer, MD, PhD is an Associate Professor and Medical Director of the McDonnel Genome Institute Clinical Sequencing Lab at Washington University in St. Louis. He is leading the SCGE Phase 2 U01 project "Genome sequencing for evaluating the efficacy, specificity, and safety of human genome editing".

12:37

Meet the Researcher: Zheng-Yi Chen

Просмотров 1662 месяца назад

Zheng-Yi Chen, D.Phil is an Associate Scientist at Mass Eye and Ear and an Associate Professor at Harvard Medical School. He is leading the SCGE Phase 2 U01 project "AAV-mediated editing to treat human autosomal dominant hearing loss DFNA41 and DFNA2".

5:58

Meet the Researcher: Nikita Kamath

Просмотров 642 месяца назад

Nikita Kamath, MS is a research associate at the Broad Institute of MIT and Harvard. She is part of the SCGE Phase 2 U19 project "Therapeutic Editing to Lower PrP in Prion Disease" led by Drs. Sonia Vallabh and Eric Minikel.

Meet the Researcher: Elizabeth Berry-Kravis

12:33

Meet the Researcher: Elizabeth Berry-Kravis

Просмотров 282 месяца назад

Elizabeth Berry-Kravis, MD, PhD, is a pediatric neurologist and professor of pediatrics and neurological sciences at Rush University Medical Center in Chicago. Dr. Berry-Kravis is a Co-PI on the SCGE Phase 2 UG3 project "A Non-viral CRISPR-mediated Genome Editing Delivery Platform As A Potential Therapy For Neurogenetic Diseases". The other Co-PIs on the project are Yong-Hui Jiang and Jiangbing...

6:28

Meet the Researcher: Lauren Testa

Просмотров 432 месяца назад

Lauren Testa is a fourth year PhD student at the University of Pennsylvania. She is part of the SCGE Phase 2 U19 project "Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases" led by Drs. William Peranteau and Kiran Musunuru.

3:56

Meet the Researcher: Marcela Tabima

Просмотров 343 месяца назад

Marcela Tabima, PhD is a Principal Scientist at the University of Wisconsin-Madison. She is part of the SCGE Phase 2 U19 project "The CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies" led by Dr. Kris Saha. Learn more about this research project at scge.mcw.edu/phase-2-ind-enabling-studies/#saha

10:22

Meet the Researcher: Ross Wilson

Просмотров 293 месяца назад

Ross Wilson, PhD is an Assistant Adjunct Professor at the University of California, Berkeley, and the Director of Therapeutic Delivery at the Innovative Genomics Institute (IGI). Dr. Wilson is part of the U19 project "Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats" led by Dr. Jennifer Dounda. Learn more about this research project at scge.mcw.edu/phase-2-i...

4:32

Meet the Researcher: Claire Clelland

Просмотров 1603 месяца назад

Claire Clelland, MD, PhD sat down to talk about the research she is doing within phase 2 of the SCGE program. Dr. Clelland is part of the U19 project "Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats" led by Dr. Jennifer Dounda. Learn more about this research project at scge.mcw.edu/phase-2-ind-enabling-studies/#doudna

4:14

SCGE Toolkit Overview

Просмотров 107 месяцев назад

The SCGE program has developed a genome editing toolkit to disseminate novel strategies, technologies, and learnings to the broader scientific community. Learn more about the toolkit and view a quick demo in this video. The toolkit can be accessed for free at scge.mcw.edu/toolkit/