Jove
Visualize
Contact Us
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

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

Biofouling on aquaculture mesh: disentangling seasonal and environmental effects with DNA metabarcoding.

Biofouling·2026
Same author

<i>In Vivo</i> Genome Editing Approach to Disrupt Hydroxyacid Oxidase 1 for the Treatment of Primary Hyperoxaluria Type 1.

Human gene therapy·2026
Same author

Rapidly progressive Riedel's thyroiditis presenting with recurrent laryngeal nerve palsy mimicking thyroid malignancy.

BMJ case reports·2026
Same author

ATF2 phosphorylation is a core transcriptional driver of neuron apoptosis.

Neuron·2026
Same author

Lung cancer-enriched p53 mutants occupy canonical p53 target genes without activating transcription, revealing a distinct loss-of-function behavior.

bioRxiv : the preprint server for biology·2026
Same author

Author Correction: Branched endosomal disruptor (BEND) lipids mediate delivery of mRNA and CRISPR-Cas9 ribonucleoprotein complex for hepatic gene editing and T cell engineering.

Nature communications·2025

Related Experiment Video

Updated: Aug 12, 2025

Combined Genetic and Chemical Capsid Modifications of Adenovirus-Based Gene Transfer Vectors for Shielding and Targeting
08:14

Combined Genetic and Chemical Capsid Modifications of Adenovirus-Based Gene Transfer Vectors for Shielding and Targeting

Published on: October 26, 2018

8.5K

Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity.

Makoto Horiuchi1, Christian J Hinderer1, Hailey N Shankle1

  • 1Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Human Gene Therapy
|January 31, 2023
PubMed
Summary

Pre-existing antibodies limit adeno-associated virus (AAV) gene therapy. A novel FcRn-inhibiting antibody, M281, reduced these neutralizing antibodies (NAbs) and enhanced AAV delivery in preclinical models.

Keywords:
FcRnadeno-associated virusgene therapyimmunityneutralizing antibody

More Related Videos

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification
04:43

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification

Published on: October 11, 2024

2.1K
Author Spotlight: Assessing Intrathecal Gene Therapy Efficacy in Juvenile Rats
04:38

Author Spotlight: Assessing Intrathecal Gene Therapy Efficacy in Juvenile Rats

Published on: March 29, 2024

1.3K

Related Experiment Videos

Last Updated: Aug 12, 2025

Combined Genetic and Chemical Capsid Modifications of Adenovirus-Based Gene Transfer Vectors for Shielding and Targeting
08:14

Combined Genetic and Chemical Capsid Modifications of Adenovirus-Based Gene Transfer Vectors for Shielding and Targeting

Published on: October 26, 2018

8.5K
Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification
04:43

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification

Published on: October 11, 2024

2.1K
Author Spotlight: Assessing Intrathecal Gene Therapy Efficacy in Juvenile Rats
04:38

Author Spotlight: Assessing Intrathecal Gene Therapy Efficacy in Juvenile Rats

Published on: March 29, 2024

1.3K

Area of Science:

  • Gene Therapy
  • Immunology
  • Pharmacology

Background:

  • Adeno-associated virus (AAV)-based gene therapies offer potential for rare genetic disorders.
  • Prevalence of anti-AAV neutralizing antibodies (NAbs) restricts patient eligibility.
  • Neonatal Fc receptor (FcRn) regulates IgG half-life, influencing NAb levels.

Purpose of the Study:

  • To evaluate the efficacy of the FcRn-inhibiting monoclonal antibody M281.
  • To assess M281's ability to reduce pre-existing anti-AAV NAbs.
  • To determine if M281 facilitates AAV gene delivery in NAb-positive subjects.

Main Methods:

  • Administration of M281 to mice and nonhuman primates.
  • Measurement of NAb titers and total IgG levels.
  • Assessment of AAV gene delivery to liver and heart post-intravenous administration.

Main Results:

  • M281 effectively reduced NAb titers and total IgG levels.
  • M281 enhanced AAV gene delivery to the liver and other organs in NAb-positive animals.
  • Successful gene delivery was observed after systemic AAV administration.

Conclusions:

  • Disrupting the FcRn-IgG interaction with M281 can mitigate pre-existing humoral immunity.
  • This approach may enable AAV-based gene therapies for NAb-positive patients.
  • FcRn inhibition presents a viable strategy to broaden AAV gene therapy accessibility.