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Results for Gene Transfer

1 - 10 of 51

Denise E. Sabatino, PhD
Denise E. Sabatino

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.

E-mail:
sabatinod [at] chop.edu
Paris Margaritis, DPhil
Paris Margaritis

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

E-mail:
margaritis [at] chop.edu
Marc Yudkoff, MD
Marc Yudkoff

Dr. Yudkoff's research is focused on the use of stable isotopes to measure the rate of production of urea in humans who have a urea cycle defect, or a congenital inability to detoxify ammonia into urea.

E-mail:
yudkoff [at] chop.edu
Hyun-Duck Nah-Cederquist, DMD, MSD, PhD
Hyun-Duck Nah-Cederquist

Dr. Nah-Cederquist investigates solutions to clinical problems in pediatric plastic surgery. Her lab is built around the strengths of CHOP's clinical practices and basic science research. This offers the unique opportunity to directly test hypotheses born from clinical problems in the laboratory, and to take new technologies and concepts developed in the laboratory to patient care.

E-mail:
nah [at] chop.edu
Colin Conine, PhD
Colin C. Conine

Dr. Conine works to understand the functions of small RNAs in reproduction, epigenetic inheritance, and development. His research focuses on how small RNAs in sperm transmit epigenetic information to offspring, as well as their involvement in male fertility.

E-mail:
conine [at] chop.edu
Laura Breda, PhD
Laura Breda

Dr. Breda is developing safe, potentially curative genetic therapies for sickle cell disease and b-thalassemia.

E-mail:
bredal [at] chop.edu

From ‘Generation to Generation:’ Faculty Spotlight With Itzhak Nissim, PhD

Published on
Apr 27, 2022
in Cornerstone Blog
Faculty Spotlight With Itzhak Nissim, PhD
Dr. Nissim studies the relationship of the metabolome and the fluxome to the genome in normal and disease states.

Research Shows Multiyear Factor VIII Expression After Hemophilia A Gene Therapy

Published on
Nov 19, 2021
in Cornerstone Blog
A one-time treatment with an adeno-associated viral vector demonstrated safe, sustained factor VIII expression for patients with hemophilia A.
Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.

Where Discovery Leads: Building on Nature's Delivery System to Advance Gene Therapy

Published on
Nov 5, 2021
in Cornerstone Blog
Raymond G. Perelman Center for Cellular and Molecular Therapeutics
The Clinical Vector Core, an award-winning, state-of-the-art, multi-product GMP manufacturing facility, supports the growing field of gene therapy.

Science Center Research Acceleration Initiative

Published on
Feb 5, 2020
in Announcements
Science Center Funding Announcement
CSL-Science Center Research Acceleration Initiative is awarding three proposals of $200,000 per year for up to two years (max $400,000 funding).
1 - 10 of 51

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In this section

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  • Principal Investigator / Faculty (5)
  • External (1)
  • Researcher / Lab Personnel (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (14)
  • Division of Hematology (5)
  • Department of Pediatrics (4)
  • Division of Neurology (3)
  • Clinical Vector Core (2)
  • Cancer Immunotherapy (CAR-T) (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Autism Research (1)
  • Center for Mitochondrial and Epigenomic Medicine (1)
  • Center for Pediatric Clinical Effectiveness (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical In Vivo Gene Therapy (1)
  • Department of Biomedical and Health Informatics (1)
  • Division of Allergy and Immunology (1)
  • Division of Oncology (1)
  • Division of Urology (1)
  • Nissim Laboratory (1)
  • Office of Academic Training and Outreach Programs (1)
  • Office of Research Safety (1)
  • Office of Technology Transfer (1)
  • Rivella Laboratory (1)

In this section

Filter By

  • Principal Investigator / Faculty (5)
  • External (1)
  • Researcher / Lab Personnel (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (14)
  • Division of Hematology (5)
  • Department of Pediatrics (4)
  • Division of Neurology (3)
  • Clinical Vector Core (2)
  • Cancer Immunotherapy (CAR-T) (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Autism Research (1)
  • Center for Mitochondrial and Epigenomic Medicine (1)
  • Center for Pediatric Clinical Effectiveness (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical In Vivo Gene Therapy (1)
  • Department of Biomedical and Health Informatics (1)
  • Division of Allergy and Immunology (1)
  • Division of Oncology (1)
  • Division of Urology (1)
  • Nissim Laboratory (1)
  • Office of Academic Training and Outreach Programs (1)
  • Office of Research Safety (1)
  • Office of Technology Transfer (1)
  • Rivella Laboratory (1)
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