Related Experiment Video

Updated: Jan 14, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

8.1K

Author Correction: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

Defne A Amado1,2, Ashley B Robbins3,4, Katherine R Whiteman3

  • 1Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. defne@pennmedicine.upenn.edu.

Nature Communications
|October 23, 2025
PubMed
Abstract

No abstract available in PubMed .

More Related Videos

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

1.8K
Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice
05:55

Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice

Published on: October 3, 2011

63.0K

Related Experiment Videos

Last Updated: Jan 14, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

8.1K
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

1.8K
Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice
05:55

Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice

Published on: October 3, 2011

63.0K

Related Concept Videos

Experimental RNAi02:15

Experimental RNAi

7.3K
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
7.3K

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

Ad5/52s fiber substitution alters Ad5 tropism, bioavailability, and plasma sensitivity.

Molecular therapy. Advances·2026

Mechanistic Insights into Structurally Tailored WEAX-Starch Interactions: Differential Inhibition of A- and B-Wheat Starch Digestion.

Journal of agricultural and food chemistry·2026

Dissecting the relationship between haplotypes around ATXN2 CAG repeats and the number of CAA interruptions by long-read sequencing.

medRxiv : the preprint server for health sciences·2026

Investigation on Fatigue Damage Characteristics of Basalt Fiber-Reinforced Asphalt Mixtures with High RAP Content.

Materials (Basel, Switzerland)·2026

Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder.

Molecular psychiatry·2026

Temporal single-cell atlas of full-length Huntington's disease mouse model defines stage-specific signatures of corticostriatal dysfunction.

Molecular neurodegeneration·2026

Human-specific morphoregulatory signatures in basal radial glia characterise neocortex evolution.

Nature communications·2026

Magnetic Electroluminescent response as a predictive indicator of operational lifetime in phosphorescent blue OLEDs.

Nature communications·2026

Genome reorganisation and expansion shape 3D genome architecture and define a distinct regulatory landscape in coleoid cephalopods.

Nature communications·2026

PI3Kδ is selectively inhibited by roginolisib by stabilizing the C-terminal helix kα12.

Nature communications·2026

A co-evolved peptide-GPCR system senses host entry to drive fungal infection.

Nature communications·2026

The conformation-selective PI3Kδ inhibitor roginolisib in patients with advanced or metastatic cancer, including metastatic uveal melanoma: first-in-human clinical trial.

Nature communications·2026

The Feasibility of Using Virtual Reality With Debriefing as a Treatment Modality for Chronic Pain.

The American journal of occupational therapy : official publication of the American Occupational Therapy Association·2026

Inflammatory profiling for precision neurorehabilitation after traumatic brain injury and stroke: a narrative review.

Frontiers in neurology·2026

Potential role of vitamin B12 supplementation in neurological recovery after severe traumatic brain injury.

Nutrition (Burbank, Los Angeles County, Calif.)·2026

Surfactant protein A (SP-A) alleviates acute kidney injury by reducing macrophage recruitment and M1 polarization.

Journal of molecular histology·2026

Long-term electromyography-based virtual reality training for chronic upper-limb motor paralysis after stroke: an exploratory single-case experimental design.

Frontiers in rehabilitation sciences·2026

Tropolone and hinokitiol act against Staphylococcus aureus by concentration-dependent iron restriction or toxic iron overload.

mSphere·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