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

Gene Therapy00:59

Gene Therapy

25.3K
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...
25.3K
CRISPR01:59

CRISPR

49.8K
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...
49.8K
What is Genetic Engineering?00:49

What is Genetic Engineering?

73.9K
Overview
73.9K
RNA Editing02:23

RNA Editing

8.9K
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...
8.9K

You might also read

Related Articles

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

Sort by
Same author

Acyclic Hydrocarbon α,ω-Dienes: From Biomass and Plastic Upcycling to Carbocycles, Precision Polymers, and Cross-Linked Elastomers.

ChemSusChem·2026
Same author

Case Report: Long-term response to multimodal treatment in metastatic uveal melanoma.

Frontiers in immunology·2026
Same author

Combining Clinician Expertise with Prompt Engineering enhances Small Language Models Reliability for Cancer Entity Recognition in Electronic Health Records.

medRxiv : the preprint server for health sciences·2025
Same author

Plasma Cell Leukemia Update on Immunophenotype, Molecular Characteristics, and Therapy. The Second Part of Plasma Cell Neoplasms with Spreading in the Blood and Tissues.

Mediterranean journal of hematology and infectious diseases·2025
Same author

Comonomer Discrimination in Copolymerization of β‑Myrcene: Ethylene Inhibition, Spectators, and Soft Elastomers with Isoprene.

ACS polymers Au·2025
Same author

Plasma Cell Neoplasms with Spreading in the Blood and Tissues: Extramedullary Myeloma Disease, a Rare Aggressive Form of Multiple Myeloma (First of Two Parts).

Mediterranean journal of hematology and infectious diseases·2025

Related Experiment Video

Updated: Jun 13, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

3.5K

Therapeutic Gene Editing for Hemoglobinopathies.

Ugo Testa1, Giuseppe Leone2, Maria Domenica Cappellini3

  • 1Istituto Superiore Sanità, Roma, Italy.

Mediterranean Journal of Hematology and Infectious Diseases
|September 11, 2024
PubMed
Summary

Gene editing offers a new era for treating sickle cell disease (SCD) and beta-thalassemia (TDT). Advanced techniques like CRISPR-Cas9 and base editing aim to correct genetic defects or boost fetal hemoglobin for effective therapies.

Keywords:
Gene editingHemoglobinopathiesSickle Cell AnemiaThalassemiagene therapy

More Related Videos

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
12:04

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells

Published on: March 10, 2023

3.5K
Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models
08:14

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Published on: October 3, 2019

12.1K

Related Experiment Videos

Last Updated: Jun 13, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

3.5K
Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
12:04

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells

Published on: March 10, 2023

3.5K
Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models
08:14

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Published on: October 3, 2019

12.1K

Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) are debilitating genetic blood disorders.
  • Current treatments for SCD and TDT are limited, necessitating novel therapeutic strategies.
  • Gene therapy has emerged as a promising avenue for treating these hemoglobinopathies.

Purpose of the Study:

  • To review recent advancements in gene editing for hemoglobinopathies.
  • To highlight the potential of gene editing in treating SCD and TDT.
  • To discuss the broader implications for monoallelic hereditary diseases.

Main Methods:

  • Review of clinical studies utilizing gene therapy and gene editing for SCD and TDT.
  • Focus on lentiviral vectors and gene editing tools such as CRISPR-Cas9, TALENs, ZFNs, and base editing.
  • Analysis of approaches to induce fetal hemoglobin or correct genetic defects.

Main Results:

  • Gene editing techniques have shown significant progress in developing new treatments for hemoglobinopathies.
  • CRISPR-Cas9, TALENs, ZFNs, and base editing offer diverse strategies for therapeutic intervention.
  • These methods aim to achieve therapeutic levels of fetal hemoglobin or directly repair the causative genetic mutations.

Conclusions:

  • Gene editing represents a paradigm shift in the treatment of hemoglobinopathies.
  • It holds promise for a curative approach to SCD and TDT.
  • The success of gene editing in hemoglobinopathies may pave the way for treating other monoallelic hereditary diseases.