Maria L. Escolar

机构：University of Pittsburgh, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center · ORCID：0000-0003-1530-6777

发表论文 210 篇 · 总被引 6091 次 · h-index 42

代表论文

• Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation (2023 · Molecular Therapy — Methods & Clinical Development · 被引 29)
• Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States (2024 · Cytotherapy · 被引 26)
• Population-Based Screening of Newborns: Findings From the NBS Expansion Study (Part One) (2022 · Frontiers in Genetics · 被引 21)
• Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy (2023 · Molecular Therapy · 被引 13)
• Improved Brain Pathology and Progressive Peripheral Neuropathy in a 15 Year Old Survivor of Infantile Krabbe Disease Treated With Umbilical Cord Transplantation (2022 · Frontiers in Molecular Neuroscience · 被引 9)
• Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy (2024 · Molecular Genetics and Metabolism · 被引 8)