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 Experiment Videos

Large animal models and gene therapy.

Margret Casal1, Mark Haskins

  • 1School of Veterinary Medicine, University of Pennsylvania, Philadelphia, 19104-6051, USA.

European Journal of Human Genetics : EJHG
|December 8, 2005
PubMed
Summary
This summary is machine-generated.

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

Skin barrier, phenotypic and genotypic characterisation of autosomal recessive ichthyosis in TGM1-deficient Jack Russell Terriers and response to topical ceramide.

Veterinary dermatology·2024
Same author

Atypical granulation in neutrophils of a domestic shorthair cat.

Veterinary clinical pathology·2024
Same author

Upregulation of Sortilin, a Lysosomal Sorting Receptor, Corresponds with Reduced Bioavailability of Latent TGFβ in Mucolipidosis II Cells.

Biomolecules·2020
Same author

Cognitive Abilities of Dogs with Mucopolysaccharidosis I: Learning and Memory.

Animals : an open access journal from MDPI·2020
Same author

Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durations.

Molecular genetics and metabolism·2016
Same author

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

Molecular therapy : the journal of the American Society of Gene Therapy·2015

Gene transfer studies in mice offer proof of principle but have limitations. Large animal models are increasingly vital for human disease research due to their physiological and genetic similarities, enabling long-term, scalable therapeutic studies.

Area of Science:

  • Biomedical Research
  • Gene Therapy
  • Animal Models

Background:

  • Gene transfer experiments for inherited and acquired diseases have predominantly used mouse models for two decades.
  • Mouse models offer proof of principle for therapeutic modalities but have limitations including short experiment durations, homogenous genetic backgrounds, and imperfect representation of human diseases.
  • Naturally occurring large animal models are gaining importance for studying human genetic diseases.

Purpose of the Study:

  • To highlight the limitations of mouse models in gene transfer research.
  • To emphasize the growing importance and advantages of large animal models in biomedical research.
  • To discuss the suitability of large animals for longitudinal studies and scaling up gene therapies.

Main Methods:

Related Experiment Videos

  • Review of existing literature on gene transfer experiments in mouse models.
  • Comparison of physiological and genetic characteristics between mouse and large animal models.
  • Analysis of the benefits of large animal models for long-term and translational studies.

Main Results:

  • Mouse models have inherent limitations that may not fully translate to human disease.
  • Large animal models offer greater genetic diversity, longer lifespans for longitudinal studies, and physiological similarities to humans.
  • The size of large animals is advantageous for addressing scalability issues in gene therapy.

Conclusions:

  • While mouse models are useful for initial gene transfer studies, their limitations necessitate the use of more relevant models.
  • Large animal models provide a more accurate preclinical platform for gene therapy development due to their human-like physiology and genetic outbreeding.
  • Further utilization of large animal models is crucial for advancing gene transfer therapies for human diseases.