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

Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

2.1K
Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
2.1K
Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

270
Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
270
Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

399
Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
399
Gene Therapy00:59

Gene Therapy

25.9K
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...
25.9K
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

17.0K
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
17.0K

You might also read

Related Articles

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

Sort by
Same author

Revisiting long-standing biological questions through evolving technologies.

Bioscience trends·2026
Same author

RNA activation as a precision dosing modality: MTL-CEBPA for controlled enzyme elevation in MPS I-H.

Frontiers in medicine·2026
Same author

C/EBPβ stabilizes oxidative stress in triple-negative breast cancer cells, driving taxane resistance and immune evasion.

Molecular therapy : the journal of the American Society of Gene Therapy·2025
Same author

ASOs reduce SARS-CoV-2 viral titers in Syrian golden hamsters.

Molecular therapy. Nucleic acids·2025
Same author

A hypoxia-responsive tRNA-derived small RNA confers renal protection through RNA autophagy.

Science (New York, N.Y.)·2025
Same author

Small RNAs as therapeutic agents: From catalytic motifs to regulatory pathways.

Molecular therapy : the journal of the American Society of Gene Therapy·2025

Related Experiment Video

Updated: Sep 22, 2025

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
14:55

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System

Published on: May 12, 2013

60.8K

Stereopure oligo therapy for ALS

John J Rossi1

  • 1City of Hope, Duarte, CA, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|May 18, 2022
PubMed
Summary

No abstract available in PubMed .

More Related Videos

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

13.7K
Novel Atomic Force Microscopy Based Biopanning for Isolation of Morphology Specific Reagents against TDP-43 Variants in Amyotrophic Lateral Sclerosis
13:31

Novel Atomic Force Microscopy Based Biopanning for Isolation of Morphology Specific Reagents against TDP-43 Variants in Amyotrophic Lateral Sclerosis

Published on: February 12, 2015

8.9K

Related Experiment Videos

Last Updated: Sep 22, 2025

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
14:55

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System

Published on: May 12, 2013

60.8K
Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

13.7K
Novel Atomic Force Microscopy Based Biopanning for Isolation of Morphology Specific Reagents against TDP-43 Variants in Amyotrophic Lateral Sclerosis
13:31

Novel Atomic Force Microscopy Based Biopanning for Isolation of Morphology Specific Reagents against TDP-43 Variants in Amyotrophic Lateral Sclerosis

Published on: February 12, 2015

8.9K