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Results for AAV 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
Johannes C.M. van der Loo, PhD
Johannes C.M. van der Loo

Dr. van der Loo is an Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania and Director of the Clinical Vector Core at CHOP, focusing on manufacturing of GMP and GMP process-comparable AAV and Lentiviral vectors for preclinical and gene therapy early phase clinical studies.

E-mail:
vanderlooj [at] chop.edu

ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists

Published on
May 18, 2023
in Cornerstone Blog
ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists
Scientists from Children’s Hospital of Philadelphia attend the American Society of Cell & Gene Therapy Annual Meeting to share their expertise through posters, panel discussions, and workshops.

Optimizing Vector Delivery to Reduce Risk: Q&A With Beverly Davidson, PhD, and Misun Hwang, MD

Published on
Feb 6, 2023
in Cornerstone Blog
Gene Therapy and Radiology Team Up to Optimize Vector Delivery
Two scientists with unique skill sets are developing an alternative method for gene therapy delivery with support from a CGTC Seed Grant.

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.

Snapshot Science: How Did Researchers Resolve Adverse Symptoms that Emerged with AAV-based RNA Interference Strategies for Ataxia Therapy?

Published on
Oct 18, 2021
in Cornerstone Blog
Researchers Resolve Symptoms that Emerged with AAV-based RNAi for SCA1
Raymond G. Perelman Center for Cellular and Molecular Therapeutics team highlights that extended safety testing of AAV-based RNAi strategies is critical.

In the News: 22q, Mentoring Award, Neonatal E. coli, Gene Therapy, Siegel Award

Published on
Nov 20, 2020
in Cornerstone Blog
In the News
This week in the news: New awards for our scientists and findings for antibiotic stewardship, gene therapy, and 22q11.2 deletion syndrome.
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In this section

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Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (12)
  • Clinical In Vivo Gene Therapy (4)
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  • Clinical Vector Core (2)
  • Department of Pediatrics (2)
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  • Advancement of Hyperinsulinism Care and Research (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Childhood Cancer Research (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical Futures (1)
  • Congenital Hyperinsulinism Research Laboratory (1)
  • De León-Crutchlow Laboratory (1)
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  • Department of Radiology (1)
  • Division of Endocrinology and Diabetes (1)
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  • Division of Neonatology (1)
  • Division of Oncology (1)
  • Division of Protective Immunity (1)
  • Division of Pulmonary and Sleep Medicine (1)
  • Human Pluripotent Stem Cell Core (1)
  • PolicyLab (1)
  • Research Vector Core (1)

In this section

Filter By

  • Principal Investigator / Faculty (2)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (12)
  • Clinical In Vivo Gene Therapy (4)
  • Division of Hematology (4)
  • Division of Neurology (3)
  • Clinical Vector Core (2)
  • Department of Pediatrics (2)
  • Division of Cardiology (2)
  • Division of Human Genetics (2)
  • Advancement of Hyperinsulinism Care and Research (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Childhood Cancer Research (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical Futures (1)
  • Congenital Hyperinsulinism Research Laboratory (1)
  • De León-Crutchlow Laboratory (1)
  • Department of Biomedical and Health Informatics (1)
  • Department of Pathology and Laboratory Medicine (1)
  • Department of Radiology (1)
  • Division of Endocrinology and Diabetes (1)
  • Division of General Pediatrics (1)
  • Division of Neonatology (1)
  • Division of Oncology (1)
  • Division of Protective Immunity (1)
  • Division of Pulmonary and Sleep Medicine (1)
  • Human Pluripotent Stem Cell Core (1)
  • PolicyLab (1)
  • Research Vector Core (1)
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