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] email.chop.edu
View Beverly L. Davidson's Profile
Can Ficicioglu, MD, PhD
Can Ficicioglu
E-mail:
ficicioglu [at] email.chop.edu
View Can Ficicioglu's Profile
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] email.chop.edu
View Rebecca C. Ahrens-Nicklas's Profile

Grant Awarded to Study Gene Therapy for Disorders With Severe Brain Disease

Published on
Dec 4, 2019
in Cornerstone Blog
John Wolfe, VMD, PhD, is studying gene therapy methods to correct the brain disease of lysosomal storage disorders.
John Wolfe, VMD, PhD, receives grant to study gene therapy treatment for lysosomal storage disorders with severe disease of the brain.

In the News: Fertility After Childhood Cancer, Restoring Vision, NYAS Podcast, Obesity and Autism, Genes in Childhood Epilepsy

Published on
Nov 2, 2018
in Cornerstone Blog
CHOP Research In the News
In this week's roundup, CHOP scientists take a stem cell approach to restore vision to people who have lost key vision cells.

CHOP Scientist Shares New Grant to Restore Vision Using Stem Cell Therapy

Published on
Oct 23, 2018
in Press Releases
A CHOP researcher is working to manipulate stem cells to restore vision in genetic cases of blindness.

In the News: Brain Plasticity, Hemophilia B Gene Therapy, Stem Cells and SCD, Medical Cannabis for Autism, PA Rare Disease Advisory Council

Published on
Dec 15, 2017
in Cornerstone Blog
CHOP Research In the News
From brain plasticity to medical cannabis for use in autism, don't miss this week's roundup of research headlines from CHOP.

CHOP Expert in Metabolic Diseases Appointed to State Rare Diseases Council

Published on
Dec 4, 2017
in Press Releases
Can Ficicioglu, MD, PhD, a physician-scientist who is an expert in genetic disorders called inborn errors of metabolism, was appointed by the Pennsylvania Department of Health to a statewide Rare Diseases Advisory Council.

Targeted Gene Therapy Aimed at Devastating Childhood Disease

Published on
Nov 12, 2015
in Cornerstone Blog
When something important is missing, we often search for a replacement. After many years of looking, a team of researchers at The Children’s Hospital of Philadelphia and the University of Missouri have found a way to substitute for a missing gene linked to a relentless childhood neurodegenerative disease.

Platelet Granule Formation Gives Insight Into Rare Disease

Published on
Jul 23, 2014
in Cornerstone Blog
Cell biologists at The Children’s Hospital of Philadelphia Research Institute speculate that platelet granules hold the key to unlocking the mechanisms behind Hermansky-Pudlak syndrome.
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