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Results for Lysosomal Storage Diseases

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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
Rebecca C. Ahrens-Nicklas, MD, PhD
Rebecca C. Ahrens-Nicklas, MD, PhD

Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.

E-mail:
ahrensnicklasr [at] chop.edu
Can Ficicioglu, MD, PhD
Can Ficicioglu
E-mail:
ficicioglu [at] chop.edu
Lucas Tricoli, PhD
Lucas Tricoli

Dr. Tricoli's research involves developing a new generation of lentiviral vectors that will prove to limit toxicity and improve activity of the ARSA enzyme, extending the efficacy of and eligibility for gene therapy in metachromatic leukodystrophy, a devastating childhood lysosomal storage disease.

E-mail:
tricolil [at] chop.edu

In the News: Top Pediatrics Program for Medical Schools, First-of-Its-Kind Gene Therapy Trial, Novel Computational Tool

Published on
May 26, 2023
in Cornerstone Blog
In the News: Top Pediatrics Program for Medical Schools, Rare Disease Research, Novel Tool
In this week’s news roundup, CHOP’s Department of Pediatrics ranked No 1. in U.S. News & World Report’s ‘Best Pediatrics Programs’ for medical schools.

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.

In the News: Novel Genetic Disorder, ‘Peanut Patch,’ Neonatal Outcomes, Seed Grants

Published on
May 12, 2023
in Cornerstone Blog
In the News: Novel Disorder, ‘Peanut Patch,’ Neonatal Outcomes, Seed Grants
Learn about a novel disorder caused by rare genetic variants, the importance of global neonatal outcomes, and more, in this week’s edition of In the News.

CHOP Researchers Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth

Published on
Jul 13, 2021
in Press Releases
CHOP researchers used a vector to edit a single base mutation in Hurler Syndrome, halting progression of a disease that causes irreversible damage before birth.

Faculty Spotlight: ‘Bench to Bedside and Back’ With Rebecca Ahrens-Nicklas, MD, PhD

Published on
May 10, 2021
in Cornerstone Blog
Faculty Spotlight: ‘Bench to Bedside and Back’ With Rebecca Ahrens-Nicklas, MD, PhD
Meet our featured Faculty Spotlight scientist for May, Rebecca Ahrens-Nicklas, MD, PhD, assistant professor of Pediatrics at CHOP.

In The News: COVID-19 Resurgence, Injury Prevention Counseling, Whole Brain Gene Therapy, Children with SARS-CoV-2 Infection, Pulse Oximetry Monitoring

Published on
Jul 31, 2020
in Cornerstone Blog
Our Scientists’ Research Highlighted In the News
This week’s roundup features projections of the spread of COVID-19 and novel findings on gene therapy to treat the whole brain. This and more In The News.
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In this section

Filter By

  • Principal Investigator / Faculty (3)
  • Research Administration (1)
  • Researcher / Lab Personnel (1)

Organizations

  • Department of Pediatrics (6)
  • Division of Human Genetics (4)
  • Division of Neurology (4)
  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (3)
  • Division of General Pediatrics (2)
  • Division of Infectious Diseases (2)
  • Division of Urology (2)
  • Research in the Department of Child and Adolescent Psychiatry and Behavioral Services (2)
  • Ahrens-Nicklas Laboratory (1)
  • Cell and Gene Therapy Collaborative (1)
  • Center for Autism Research (1)
  • Center for Childhood Cancer Research (1)
  • Center for Computational and Genomic Medicine (1)
  • Center for Injury Research and Prevention (1)
  • Department of Biomedical and Health Informatics (1)
  • Division of Allergy and Immunology (1)
  • Division of Cardiology (1)
  • Division of Hematology (1)
  • Division of Neonatology (1)
  • Intellectual and Developmental Disabilities Research Center (1)
  • Neonatal Chronic Lung Disease (1)
  • Neuroscience Research Affinity Group (1)
  • Pennsylvania Pediatric Medical Device Consortium (1)
  • Wolfe Lab (1)
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