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

RNA Editing02:23

RNA Editing

9.7K
RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
9.7K
CRISPR01:59

CRISPR

57.3K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
57.3K
Base Excision Repair01:54

Base Excision Repair

25.8K
One of the common DNA damages is the chemical alteration of single bases by alkylation, oxidation, or deamination. The altered bases cause mispairing and strand breakage during replication. This type of damage causes minimal change to the DNA double helix structure and can be repaired by the base excision repair (BER) pathways. BER corrects damaged DNA sequences by removing the damaged base and restoring the original base sequence using the complementary strand as a template.
The first step of...
25.8K

You might also read

Related Articles

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

Sort by
Same author

DNASE1L3 Deficiency With Novel Missense Variant: Enzymatic and Plasma Fragmentomic Evidence of Pathogenicity and Partial Response to JAK Blockade.

ACR open rheumatology·2026
Same author

Variant profile of Brazilian patients with Sanfilippo syndrome type B.

Genetics and molecular biology·2026
Same author

Publisher Correction: Biallelic variants in RNU2-2 cause the most prevalent known recessive neurodevelopmental disorder.

Nature genetics·2026
Same author

Genetic insights into the peoples who shaped the American continent.

Genetics and molecular biology·2026
Same author

Biallelic variants in RNU2-2 cause the most prevalent known recessive neurodevelopmental disorder.

Nature genetics·2026
Same author

Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I.

Pharmaceutics·2026

Related Experiment Video

Updated: Dec 30, 2025

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
09:37

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development

Published on: March 5, 2017

13.5K

Genome Editing for Mucopolysaccharidoses.

Edina Poletto1,2, Guilherme Baldo1,2, Natalia Gomez-Ospina3

  • 1Gene Therapy Center, Hospital de Clinicas de Porto Alegre, Porto Alegre 90035-007, Brazil.

International Journal of Molecular Sciences
|January 17, 2020
PubMed
Summary

Genome editing offers potential curative therapies for genetic diseases like mucopolysaccharidoses (MPS). Current research shows promise in preclinical and clinical studies, paving the way for future MPS treatments.

Keywords:
CRISPR/Cas9HunterHurlerZinc Finger Nucleasesgene therapygenome editinghematopoietic stem cell transplantationlysosomal storage diseasemucopolysaccharidosesnon-viral vectorsviral vectors

More Related Videos

Modeling Mitochondrial Disease Using Brain Organoids: A Focus on Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes
08:56

Modeling Mitochondrial Disease Using Brain Organoids: A Focus on Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes

Published on: October 10, 2025

449
A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations
08:22

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations

Published on: December 1, 2017

8.9K

Related Experiment Videos

Last Updated: Dec 30, 2025

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
09:37

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development

Published on: March 5, 2017

13.5K
Modeling Mitochondrial Disease Using Brain Organoids: A Focus on Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes
08:56

Modeling Mitochondrial Disease Using Brain Organoids: A Focus on Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes

Published on: October 10, 2025

449
A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations
08:22

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations

Published on: December 1, 2017

8.9K

Area of Science:

  • Biotechnology
  • Genetics
  • Medical Research

Background:

  • Genetic diseases, particularly mucopolysaccharidoses (MPS), present significant challenges.
  • The pathophysiology of MPS is amenable to correction through various therapeutic strategies.

Purpose of the Study:

  • To review current preclinical and clinical studies on genome editing for MPS.
  • To highlight the potential of novel genome editing platforms for treating MPS.

Main Methods:

  • Review of existing literature on genome editing applications in MPS.
  • Analysis of preclinical and clinical development studies for MPS I and MPS II.

Main Results:

  • In vivo genome editing approaches have reached clinical testing for MPS I and MPS II.
  • Established therapeutic tools for MPS I and II are expected to benefit other MPS types.

Conclusions:

  • Genome editing presents a promising therapeutic avenue for mucopolysaccharidoses.
  • Advancements in genome editing platforms offer expanded therapeutic potential beyond current approaches.
  • Future strategies will likely involve multiple genome editing approaches for individual MPS diseases.