Related Experiment Video

Updated: Mar 7, 2026

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

4.5K

Genome Editing for Sickle Cell Disease: A Little BCL11A Goes a Long Way

Mir A Hossain1, Jörg Bungert1

  • 1Department of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL 32610, USA; UF Health Cancer Center, Center for Epigenetics, Genetics Institute, University of Florida, Gainesville, FL 32610, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|February 14, 2017
PubMed
Abstract

No abstract available in PubMed .

More Related Videos

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

13.1K
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

5.0K

Related Experiment Videos

Last Updated: Mar 7, 2026

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

4.5K
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

13.1K
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

5.0K

Related Concept Videos

CRISPR01:59

CRISPR

58.5K
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...
58.5K
iPS Cell Differentiation01:22

iPS Cell Differentiation

3.2K
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
3.2K

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

Epithelial-Mesenchymal Transition Activates YAP to Drive Malignant Progression and Immune Evasion.

Cancers·2025

TFII-I/GTF2I regulates globin gene expression and stress response in erythroid cells.

The Journal of biological chemistry·2025

Enhancer-Mediated Formation of Nuclear Transcription Initiation Domains.

International journal of molecular sciences·2022

Regulation of RNA Polymerase II Transcription Initiation and Elongation by Transcription Factor TFII-I.

Frontiers in molecular biosciences·2021

Post-translational modification of RNA m6A demethylase ALKBH5 regulates ROS-induced DNA damage response.

Nucleic acids research·2021

Super-enhancer mediated regulation of adult β-globin gene expression: the role of eRNA and Integrator.

Nucleic acids research·2021

Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

A phase 1/2 dose-escalation study of sepiapterin in patients with 6-pyruvoyl-tetrahydropterin synthase deficiency with hyperphenylalaninemia.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

D-Amino Acid Prodrug DLMEH Activates mTORC1 via Sestrin2 to Restore Muscle Protein Synthesis in Sarcopenia.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Combination Of Bispecific Antibodies Enable Targeted TNFRSF Agonism and Effective Antitumor Activity.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Protein-based masks to unlock conditional activation of therapeutics.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Optimization of AsCas12f1-Mediated Long-Term Gene Repression.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Successful management of anastomotic false aneurysm after a femoro-popliteal bypass with percutaneous injection of fibrin sealant.

Jornal vascular brasileiro·2026

A biomimetic core-shell nanofibrous dressing for temporally coordinated infection control and mitochondrial protection in diabetic wounds.

Materials today. Bio·2026

Rethinking osteocyte models in vitro: the need for mineralised extracellular matrix and network architecture.

Frontiers in bioengineering and biotechnology·2026

Experience of biological implants in thoracic surgery for soft tissue and chest wall reconstruction in clean and contaminated environments.

Journal of thoracic disease·2026

Bioinductive collagen scaffolds for partial- and full-thickness rotator cuff repair: a systematic review.

Clinics in shoulder and elbow·2026

A Biomimetic Nasal Mucus Gel System Adaptable for Catalytic Antibacterial Therapy.

Small (Weinheim an der Bergstrasse, Germany)·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