Thomas Weber

Spark Therapeutics (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

47h-index9.7kcitations147works6.52yr avg
OpenAlex↗ORCID↗Google Scholar↗Lab page↗

Accepting Students?

No reports yet. Know if this professor is taking students?

Research Topics

Virus-based gene therapy research(38), Viral Infections and Immunology Research(18), Viral Infectious Diseases and Gene Expression in Insects(17), Lipid Membrane Structure and Behavior(15), Cellular transport and secretion(15)

Publications147 total

Systemic Administration of AAV9-G266A with Focused Ultrasound Enables Robust Brain Transduction with Minimal Liver Transduction.
PubMed·2026
Ethics in Corrosion
2025
Human Ethics in Corrosion
2025
Correction: Lopez-Gordo et al. Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights. Viruses 2024, 16, 442
Viruses·2024Open Access
NAVIGATING THE UNKNOWN: HOW HEALTHCARE ENTREPRENEURS MANAGE UNCERTAINTY
Journal of Developmental Entrepreneurship·2024· 1 cited
Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights
Viruses·2024· 38 citedOpen Access
Selective Anti-AAV Antibody Depletion by Hemapheresis and Immunoadsorption
Methods in molecular biology·2022· 2 citedOpen Access
Hydroxylation of N-acetylneuraminic Acid Influences the in vivo Tropism of N-linked Sialic Acid-Binding Adeno-Associated Viruses AAV1, AAV5, and AAV6
Frontiers in Medicine·2021· 5 citedOpen Access
Abstract 10248: Regulation of the Methylation and Expression Levels of the Bmpr2 Gene by Sin3a as a Novel Therapeutic Mechanism in Pulmonary Arterial Hypertension
Circulation·2021
Abstract 10253: Sin3a Gene Delivery Restores Bmpr2 Expression Through Foxk2 in Pulmonary Arterial Endothelial Cells and Inhibits Sugen/Hypoxia-Induced Pah
Circulation·2021
Regulation of the Methylation and Expression Levels of the BMPR2 Gene by SIN3a as a Novel Therapeutic Mechanism in Pulmonary Arterial Hypertension
Circulation·2021· 87 citedOpen Access
Renale Sympathikusdenervierung – Neue Aspekte 2021
Journal für Kardiologie (Krause & Pachernegg GmbH)·2021Open Access
Correction: Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
Gene Therapy·2021· 7 citedOpen Access
Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions
Frontiers in Immunology·2021· 147 citedOpen Access
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
Gene Therapy·2021· 104 citedOpen Access
Abstract 13932: Lung-targeted Sin3a Gene Therapy as a Promising Strategy to Restore Bmpr2 Expression in Pulmonary Arterial Hypertension
Circulation·2020
Identification of Genes and Pathways Regulated by Lamin A in Heart
Journal of the American Heart Association·2020· 22 citedOpen Access
Intratracheal Gene Delivery of SIN3A Inhibits Pulmonary Arterial Hypertension and Restores BMPR2 Expression
2020Open Access
Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption
Molecular Therapy — Methods & Clinical Development·2020· 71 citedOpen Access
3213 Unraveling the role of Phospholamban (PLN) in humans via the characterization of Induced Pluripotent Stem Cell (iPSC) Cardiomyocytes (CM) derived from carriers of a lethal PLN mutation
Journal of Clinical and Translational Science·2019Open Access
page 1 of 8Next →

Frequent Co-authors

James A. McNew(9), Roger J. Hajjar(8), Thomas Söllner(7), James E. Rothman(7), Francesco Parlati(6), Kiyotake Ishikawa(5), Mathieu Nonnenmacher(4), Benedikt Westermann(3), Nikolaos I. Stilianakis(3), Robert J. Johnston(3), Thomas J. Melia(3), Kleopatra Rapti(3), Erik Kohlbrenner(3), Jalish M. Riyad(3), Josef Brunner(3), Kenneth Fish(3), Thomas J. Bach(3), Walter Nickel(2), Lisa Tilemann(2), Kyle Chamberlain(2)