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The Horae Gene Therapy Center
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Cystic Fibrosis
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The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium
Intrathecal gene therapy for neurologic disease in humans
Special Editorial Feature: Learning from Our Failures
Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies
ESGCT 2022: The New Normal for the Gene Therapy Community
It Is Time to Call Anti-Asian Bias What It Is: Racism
Women Lead Gene Therapy Science in 2021
CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing
Gene and Cell Therapy for Inherited and Acquired Immune Deficiency
Science Policy and Funding Implications of the 2020 U.S. Election
Black Lives Matter to Gene Therapy
Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
Barrie J. Carter: The Science and the Scientist
Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors
A Message to Our Community in the Midst of the COVID-19 Pandemic
Impact on Women's Health: Gene Therapy in Gynecologic Oncology
Free Gene and Cell Therapy Services Offered by the National Heart, Lung, and Blood Institute of the National Institutes of Health
2020: Gene Therapy Enters Its Fourth Decade
Ringing in the Changes
Prime Editing: A Novel Cas9-Reverse Transcriptase Fusion May Revolutionize Genome Editing
Epigenome Editing Strategies for Low Back Pain
Getting Tough on Capsid Screening: Tough Decoys Enable Barcoding of Vectors Capable of both Entry and Expression
Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing
New Horizons for Immune Gene Therapy
Top Five Gene Therapy Stories of 2019
DNA Vaccination in 2018: An Update
Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing
Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy
Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach
Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century : 21
Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First
Gene and Cell Therapy in 2018: A Look Ahead
Therapeutic Advances in Germany and Beyond
The Renaissance of Gene and Cell Therapy: Florence 2016
AAV Is Now a Medicine: We Had Better Get This Right
The Negative Effects of Immigration Restrictions on the Gene Therapy Community
Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy?
The Science Policy Implications of a Trump Presidency
Gene Drives: Biological Shield or Ecological Menace?
The Target's the Thing
Why Human Gene Therapy Scientists Should Care About Model Organisms
A Gene Therapy Scientist's Life Well-Lived
Special Note from the Editor-in-Chief
The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy
Transcription Activator-Like Nucleases Enable Allogeneic Chimeric Antigen Receptor-T Cell Therapy in Humans
Ethical Implications of the Cost of Molecularly Targeted Therapies
The End of the Beginning of Gene Therapy
Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question?
Autoimmunity in a genetic disease—a cautionary tale
Recent developments in the protection of pediatric research subjects
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Research
Research
Cystic Fibrosis
Alpha-1-Antitrypsin Deficiency
Fatty Oxidation Disorders
Adeno-associated virus vectors (AAVs)
Lab Members
Lab Members
Principal Investigator
Associated Faculty
Current Lab Members
Former Lab Members
Publications
Publications
Journal Articles
Reviews
Editorials
Partner with Us
Contact Us
Donate
Additional Resources