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

27.6K
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...
27.6K
Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy01:30

Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy

738
Various diagnostic tests are employed in the diagnostic process for Inflammatory Bowel Disease (IBD), particularly to differentiate between Crohn's disease and ulcerative colitis.
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...
738
Effect of Hepatic Disease on Pharmacokinetics: Drug Dosing and Hepatic Blood Flow01:26

Effect of Hepatic Disease on Pharmacokinetics: Drug Dosing and Hepatic Blood Flow

244
Chronic liver disease significantly impacts drug metabolism due to alterations in hepatic blood flow and enzyme accessibility. This disruption affects the body's pharmacokinetics—the movement and processing of drugs within the system. Key enzymes crucial for metabolizing medications become less accessible, changing how drugs are processed and utilized. Furthermore, liver disease influences the synthesis of plasma proteins, such as albumin and globulins, which play critical roles in drug...
244
Group Therapy01:26

Group Therapy

434
Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
434
Gene Flow02:39

Gene Flow

37.8K
Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
37.8K
Gene Conversion02:08

Gene Conversion

10.6K
Other than maintaining genome stability via DNA repair, homologous recombination plays an important role in diversifying the genome. In fact, the recombination of sequences forms the molecular basis of genomic evolution. Random and non-random permutations of genomic sequences create a library of new amalgamated sequences. These newly formed genomes can determine the fitness and survival of cells. In bacteria, homologous and non-homologous types of recombination lead to the evolution of new...
10.6K

You might also read

Related Articles

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

Sort by
Same author

Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report.

Journal of human immunity·2026
Same author

Response to "Defining durability in SCD gene therapy".

Blood advances·2026
Same author

Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease.

Blood·2026
Same author

Clinical Outcomes of Lentiviral Vector Gene Therapy for Sickle Cell Disease.

Blood advances·2026
Same author

A blueprint to accelerate rare pediatric gene therapy approvals.

Nature medicine·2026
Same author

Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study.

Journal of human immunity·2026

Related Experiment Video

Updated: Jan 31, 2026

Ole Isacson: Development of New Therapies for Parkinson's Disease
23:53

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

8.4K

Gene therapy for blood diseases.

Donald B Kohn1

  • 1Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, David Geffen School of Medicine, USA; Department of Pediatrics, University of California, Los Angeles, David Geffen School of Medicine, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, David Geffen School of Medicine, USA.

Current Opinion in Biotechnology
|January 2, 2019
PubMed
Summary
This summary is machine-generated.

Gene therapy offers new treatments for genetic blood disorders like hemophilia and sickle cell disease using gene addition or editing. This approach is increasingly successful, with several therapies now licensed and more in development.

More Related Videos

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

4.4K
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
15:48

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

10.1K

Related Experiment Videos

Last Updated: Jan 31, 2026

Ole Isacson: Development of New Therapies for Parkinson's Disease
23:53

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

8.4K
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

4.4K
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
15:48

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

10.1K

Area of Science:

  • Hematology
  • Molecular Biology
  • Genetic Medicine

Background:

  • Genetic diseases impact blood proteins and cells, necessitating novel therapeutic strategies.
  • Gene therapy, utilizing gene addition or editing, presents a promising avenue for treating these conditions.

Purpose of the Study:

  • To review the current state and advancements in gene therapy for blood-related genetic disorders.
  • To highlight the progress and future scope of gene therapy in clinical practice.

Main Methods:

  • In vivo gene delivery using adeno-associated virus (AAV) vectors for protein deficiencies.
  • Ex vivo gene addition or editing of hematopoietic stem cells or T cells for blood cell diseases.

Main Results:

  • Gene therapy is demonstrating routine clinical benefit for various genetic blood disorders.
  • Several gene therapies have received regulatory approval in the US and EU.
  • Ongoing advancements are expanding the therapeutic applications of gene therapy.

Conclusions:

  • Gene therapy is a rapidly advancing field with significant clinical impact on genetic blood diseases.
  • The continued development of gene therapy techniques promises broader applications and improved patient outcomes.