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Results for Viral Vectors

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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
Matthew D. Weitzman, PhD
Matthew D. Weitzman Headshot

Dr. Weitzman's research program aims to understand host responses to virus infection, and the cellular environment encountered and manipulated by viruses. He studies multiple viruses in an integrated experimental approach that combines biochemistry, molecular biology, genetics, and cell biology.

E-mail:
weitzmanm [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
Beverly L. Davidson, PhD
Photo of Beverly L. Davidson, PhD

Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.

E-mail:
davidsonbl [at] chop.edu
Perry Demsko
Perry Demsko

Perry Demsko's current research projects include in-vivo gene therapies for diseases of the hematopoietic lineage and novel viral pseudotypes.

E-mail:
demskop1 [at] chop.edu

Celebrating the Unsung Heroes of Research: The Ninth Annual CRC RE@CH Awards

Published on
May 19, 2022
in Cornerstone Blog
The Ninth Annual CRC RE@CH Award Winners Named
The Ninth Annual CRC RE@CH Awards celebrated all clinical research staff at CHOP for their contributions to the research community with a spotlight on two honorees.

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.

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.

Tunable Switch Via Oral Small Molecule Controls Gene Therapy Expression Levels

Published on
Jul 26, 2021
in Cornerstone Blog
Tunable Switch Via Oral Small Molecule Controls Gene Therapy Expression Levels  Oral Small Molecule Helps to Fine-Tune Gene Therapy Expression
Children’s Hospital of Philadelphia scientists developed a tunable switch system to control levels of gene therapy expression.
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In this section

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  • Principal Investigator / Faculty (4)
  • Research Administration (1)
  • Researcher / Lab Personnel (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (19)
  • Research Vector Core (7)
  • Division of Hematology (6)
  • Department of Pediatrics (5)
  • Clinical In Vivo Gene Therapy (3)
  • Department of Pathology and Laboratory Medicine (3)
  • Division of Neurology (3)
  • Center for Injury Research and Prevention (2)
  • Division of General Pediatrics (2)
  • Division of Infectious Diseases (2)
  • Division of Oncology (2)
  • Advancement of Hyperinsulinism Care and Research (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Applied Genomics (1)
  • Center for Childhood Cancer Research (1)
  • Center for Computational and Genomic Medicine (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical Futures (1)
  • Clinical Vector Core (1)
  • Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (1)
  • Congenital Hyperinsulinism Research Laboratory (1)
  • Davidson Laboratory (1)
  • De León-Crutchlow Laboratory (1)
  • Department of Anesthesiology and Critical Care Medicine (1)
  • Department of Biomedical and Health Informatics (1)
  • Department of Surgery (1)
  • Division of Cardiology (1)
  • Division of Endocrinology and Diabetes (1)
  • Division of Human Genetics (1)
  • Division of Pulmonary and Sleep Medicine (1)
  • Lifespan Brain Institute (1)
  • PolicyLab (1)
  • Research in the Department of Child and Adolescent Psychiatry and Behavioral Services (1)
  • Rivella Laboratory (1)
  • Wolfe Lab (1)

In this section

Filter By

  • Principal Investigator / Faculty (4)
  • Research Administration (1)
  • Researcher / Lab Personnel (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (19)
  • Research Vector Core (7)
  • Division of Hematology (6)
  • Department of Pediatrics (5)
  • Clinical In Vivo Gene Therapy (3)
  • Department of Pathology and Laboratory Medicine (3)
  • Division of Neurology (3)
  • Center for Injury Research and Prevention (2)
  • Division of General Pediatrics (2)
  • Division of Infectious Diseases (2)
  • Division of Oncology (2)
  • Advancement of Hyperinsulinism Care and Research (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Applied Genomics (1)
  • Center for Childhood Cancer Research (1)
  • Center for Computational and Genomic Medicine (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical Futures (1)
  • Clinical Vector Core (1)
  • Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (1)
  • Congenital Hyperinsulinism Research Laboratory (1)
  • Davidson Laboratory (1)
  • De León-Crutchlow Laboratory (1)
  • Department of Anesthesiology and Critical Care Medicine (1)
  • Department of Biomedical and Health Informatics (1)
  • Department of Surgery (1)
  • Division of Cardiology (1)
  • Division of Endocrinology and Diabetes (1)
  • Division of Human Genetics (1)
  • Division of Pulmonary and Sleep Medicine (1)
  • Lifespan Brain Institute (1)
  • PolicyLab (1)
  • Research in the Department of Child and Adolescent Psychiatry and Behavioral Services (1)
  • Rivella Laboratory (1)
  • Wolfe Lab (1)
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