Related Experiment Video

Updated: Jun 5, 2025

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
07:43

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices

Published on: July 14, 2023

1.8K

Imaging readiness in the gene therapy era-exploring standardized protocols for response assessment

Asthik Biswas1, Audrey K S Soo2,3, Manju A Kurian2,3

  • 1Department of Neuroradiology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

Journal of Inherited Metabolic Disease
|December 9, 2024
PubMed
Summary

No abstract available in PubMed .

More Related Videos

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells
06:47

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

Published on: October 6, 2023

461
Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids
08:52

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids

Published on: November 22, 2021

3.9K

Related Experiment Videos

Last Updated: Jun 5, 2025

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
07:43

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices

Published on: July 14, 2023

1.8K
A GMP-Compliant Procedure for the Generation of Gene-Modified T cells
06:47

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

Published on: October 6, 2023

461
Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids
08:52

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids

Published on: November 22, 2021

3.9K

Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.2K
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...
25.2K

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

Peri-ictal magnetic resonance imaging findings in pediatric seizures: a scoping review on pearls and pitfalls from a heterogeneous population.

Translational pediatrics·2026

AI integration in pediatric radiology: perspectives from international academic leaders.

European radiology·2026

Comprehensive Structural MRI Phenotyping in Oligophrenin 1-Related Disorder Reveals Characteristic Brain Malformations.

AJNR. American journal of neuroradiology·2026

ATP6V1B1-A Novel Genetic Association Between Pendred Imaging Phenotype and Renal Tubular Acidosis.

The Laryngoscope·2026

Visual morbidity, long-term outcome and prognostic factors in infants and young children with optic pathway low-grade glioma.

Neuro-oncology practice·2026

A prognostic human brain network for diffuse midline glioma.

Nature·2026

High-Protein Diet Ameliorates Cardiomyopathy in a Cardiac-Specific AGL Knockout Mouse Model: Association With Upregulated Hepatic Gluconeogenesis.

Journal of inherited metabolic disease·2026

Behavioral Phenotyping of the Pahenu2 Mouse Model for Phenylketonuria-A Scoping Review and Future Perspectives.

Journal of inherited metabolic disease·2026

Baat-Deficient Mice Recapitulate Elevated 7α-Hydroxy-3-Oxo-4-Cholestenoic Acid Observed in a Japanese Patient With BAAT Deficiency.

Journal of inherited metabolic disease·2026

Creatine Supplementation Reduces Guanidinoacetate via Downregulation of AGAT in a Mouse Model of GAMT Deficiency.

Journal of inherited metabolic disease·2026

Hepatic Glycogen Storage Disease Type IX: Long-Term Outcomes in the UK From 89 Patients.

Journal of inherited metabolic disease·2026

Liver Cancer in Methylmalonic and Propionic Acidemias: A Rare Complication? A Clinico-Pathological Study of 24 Livers.

Journal of inherited metabolic disease·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