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

Optimizing Cardiac Delivery of Modified mRNA.

Nishat Sultana1, Ajit Magadum1, Yoav Hadas1

  • 1Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|April 9, 2017
PubMed
Summary

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

The impact of an ambulance destination policy change on emergency department care metrics in New York City.

The American journal of emergency medicine·2026
Same author

Microenvironmental CTHRC1 has a pro-tumorigenic role in colorectal cancer.

Oncotarget·2026
Same author

Reprogramming the diseased liver: antioxidant-engineered mRNA nanoparticles as microenvironment modulators in MAFLD.

Immunometabolism (Cobham, Surrey)·2026
Same author

Sound-evoked auditory neurophysiological signals are a window into prodromal functional differences in a preclinical model of Alzheimer's disease.

Scientific reports·2026
Same author

Patterns of penetrating trauma in New York City before, during, and after the COVID-19 pandemic.

Journal of trauma and injury·2026
Same author

Influenza hijacks myeloid cells to inflict type-I interferon-fueled damage in the heart.

Immunity·2026

Optimizing modified mRNA (modRNA) delivery to the heart requires specific nucleotide modifications and optimized reagent concentrations. These findings advance modRNA gene therapy for cardiovascular diseases.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Cardiovascular Research

Background:

  • Modified mRNA (modRNA) is a promising somatic gene transfer technology for cardiac applications.
  • Previous studies demonstrate modRNA's potential for protein expression and improving outcomes in cardiac injury models.
  • Optimal modRNA formulations and delivery reagents for cardiac gene transfer remain uncharacterized.

Purpose of the Study:

  • To elucidate optimal parameters for cardiac modRNA delivery.
  • To investigate the impact of nucleotide modifications, modRNA doses, and transfection reagents.
  • To optimize modRNA expression in cardiac cells and tissue both in vitro and in vivo.

Main Methods:

  • Systematic testing of different nucleotide modifications, including N1-Methylpseudouridine-5'-Triphosphate.
Keywords:
deliveryheartmodified mRNA

Related Experiment Videos

  • Evaluation of varying modRNA doses and transfection reagents in cardiac cells (in vitro).
  • Assessment of modRNA delivery in mouse hearts using sucrose-citrate buffer (in vivo).

    • Main Results:

    • N1-Methylpseudouridine-5'-Triphosphate identified as the optimal nucleotide modification for cardiac modRNA delivery.
    • Optimal in vitro conditions: 0.013 μg modRNA/mm²/500 cardiomyocytes with a positively charged transfection reagent.
    • Optimal in vivo conditions: 100 μg modRNA per mouse heart in sucrose-citrate buffer.

    Conclusions:

    • Optimized conditions for in vitro and in vivo cardiac modRNA delivery have been established.
    • The identified parameters facilitate efficient gene delivery using modRNA in cardiac settings.
    • These findings support the use of modRNA-based gene therapy in various cardiovascular disease models.