Related Experiment Video

Updated: Nov 20, 2025

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
06:10

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

691

Considerations for Systemic Use of Gene Therapy

Barry J Byrne1, Manuela Corti1, Francesco Muntoni2

  • 1Powell Gene Therapy Center and Child Health Research Institute, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|January 24, 2021
PubMed
Summary

No abstract available in PubMed .

More Related Videos

Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications
03:52

Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

Published on: February 9, 2024

3.3K
Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
07:43

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices

Published on: July 14, 2023

2.7K

Related Experiment Videos

Last Updated: Nov 20, 2025

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
06:10

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

691
Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications
03:52

Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

Published on: February 9, 2024

3.3K
Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
07:43

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices

Published on: July 14, 2023

2.7K

Related Concept Videos

Gene Therapy00:59

Gene Therapy

26.8K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
26.8K
What is Genetic Engineering?00:49

What is Genetic Engineering?

77.3K
Overview
77.3K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

668
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
668

Articles linked to this work by shared authors, journal, and citation graph.

Meeting report: 2025 muscular dystrophy association summit on 'safety and challenges in gene therapy of neuromuscular diseases'.

Journal of neuromuscular diseases·2026

Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophy.

Journal of neuromuscular diseases·2026

Gait analysis reveals new outcome measures for monitoring disease progression in individuals with late-onset Pompe disease.

Journal of neuroengineering and rehabilitation·2026

Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease.

Therapeutic advances in rare disease·2026

Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRI.

Translational research : the journal of laboratory and clinical medicine·2026

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series.

Advances in therapy·2026

Addressing the package: Cell-specific gene delivery using lentiviral vectors.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

DDX3X overexpression in mice can cause rapid tissue-specific toxicity and mortality.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Multivalent DNA vaccines induce potent cellular responses and prolong BCG-mediated control of Mycobacterium tuberculosis.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Recent Progress in the Rational Design of mRNA Vaccines.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

In vivo-directed evolution identifies AAV-WM04 as a next-generation vector for potent and sustained hearing restoration in DFNB9.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

miR-146a Mimic Therapy Protects Against Platelet-Mediated Thrombosis.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Development of a Novel, Eco-Friendly Mechanical Cell Lysis Process Using Hollow Fiber Filter for AAV Manufacturing.

Biotechnology and bioengineering·2026

Programmed repair of disease-causing UGA premature termination codons in mammalian brain.

Nucleic acids research·2026

Case Report: Novel homozygous pathogenic variant of the <i>SPG20</i> gene causes the Troyer syndrome in China.

Frontiers in genetics·2026

Molecular profiling and functional analyses of umbilical cord mesenchymal stem cell-derived extracellular vesicles for dry eye diseases.

Extracellular vesicles and circulating nucleic acids·2026

Immunotherapy for tuberculosis: current landscape, mechanistic insights, and translational perspectives.

Frontiers in immunology·2026

Real-World Effectiveness and Safety of Tildrakizumab in Patients With Moderate-to-Severe Psoriasis After Failure of Ustekinumab: A Multicenter Retrospective Study.

The Journal of clinical and aesthetic dermatology·2026
See all related articles
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies
Jove
Visualize
Contact Us