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Results for Hemophilia Gene Therapy

1 - 10 of 46

Lindsey A. George, MD
Lindsey A. George

Dr. George's basic and clinical research interests are in the development of novel therapeutics for hemophilia. Her basic science laboratory studies the molecular basis of coagulation, and she is the principal investigator of ongoing hemophilia A and B gene therapy trials.

E-mail:
georgel [at] chop.edu
Benjamin J. Samelson-Jones, MD, PhD
Benjamin J. Samelson-Jones

Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. His research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.

E-mail:
samelsonjonesb [at] chop.edu
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
Rodney M. Camire, PhD
Rodney M. Camire Headshot

Dr. Camire's research focuses on understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis. He is also interested in developing novel therapeutic approaches (protein, gene-based, small molecule) to mitigate these events, which are major causes of morbidity and mortality worldwide.

E-mail:
camire [at] chop.edu

Katherine High, MD, Awarded Esteemed CHOP Gold Medal

Published on
May 12, 2022
in Cornerstone Blog
Katherine High, MD, Awarded Esteemed CHOP Gold Medal
CHOP recognized gene therapy pioneer, Katherine High, MD, with Children’s Hospital of Philadelphia’s Gold Medal Award.

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.

CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events

Published on
Nov 17, 2021
in Press Releases
Results from multicenter study are first to show improved production of coagulation factor VIII over prolong period, reducing bleeding events.

In The News: COVID-19 Depression, Outstanding Science Award, Sports Injuries, Junior Investigator Awards, Philadelphia Magazine

Published on
May 14, 2021
in Cornerstone Blog
In the News
This week’s research news features Ran Barzilay, PhD, and the results of the latest COVID-19 Resilience Project from the Lifespan Brain Institute.

From Mentors to Making it Your Own: Inspiration From Women in STEM

Published on
Mar 8, 2021
in Cornerstone Blog
Inspiration from Women in STEM at CHOP
Nominated by their Centers of Emphasis, women in STEM share what drives them, their words of wisdom, and why they do what they do.
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In this section

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  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (15)
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In this section

Filter By

  • Principal Investigator / Faculty (5)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (15)
  • Division of Hematology (13)
  • Center for Injury Research and Prevention (8)
  • Center for Childhood Cancer Research (6)
  • Department of Pediatrics (5)
  • Center for Autism Research (4)
  • PolicyLab (4)
  • Center for Computational and Genomic Medicine (3)
  • Center for Mitochondrial and Epigenomic Medicine (3)
  • Center for Pediatric Clinical Effectiveness (3)
  • Department of Anesthesiology and Critical Care Medicine (3)
  • Department of Biomedical and Health Informatics (3)
  • Division of Oncology (3)
  • Center for Applied Genomics (2)
  • Clinical In Vivo Gene Therapy (2)
  • Division of Endocrinology and Diabetes (2)
  • Division of Human Genetics (2)
  • Division of Pulmonary and Sleep Medicine (2)
  • Mitochondrial Medicine (2)
  • PennCHOP Microbiome Program (2)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Center for Violence Prevention (1)
  • Children’s Oncology Group (1)
  • Department of Child and Adolescent Psychiatry and Behavioral Sciences (1)
  • Department of Radiology (1)
  • Division of Neonatology (1)
  • Division of Nephrology (1)
  • Division of Neurology (1)
  • Division of Neurosurgery (1)
  • Division of Protective Immunity (1)
  • Lifespan Brain Institute (1)
  • Office of Academic Training and Outreach Programs (1)
  • Pediatric Center of Excellence in Nephrology (1)
  • Pennsylvania Pediatric Medical Device Consortium (1)
  • Research Vector Core (1)
  • Stress Neurobiology Program (1)
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