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 Experiment Videos

[Gene therapy--basic principles]

H E Blum1

  • 1Departement für Innere Medizin, Universitätsspital Zürich.

Praxis
|January 10, 1995
PubMed
Summary
This summary is machine-generated.

Recombinant DNA technology enables gene cloning and in vitro expression, forming the basis of human gene therapy. This therapy uses gene replacement, augmentation, or expression blocking for genetic diseases.

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

The transcription factor c-JUN/AP-1 promotes HBV-related liver tumorigenesis in mice.

Cell death and differentiation·2015
Same author

[Stimulus-triggered acquisition of pluripotency: a new perspective in reprogramming of differentiated somatic cells].

Deutsche medizinische Wochenschrift (1946)·2014
Same author

[Sofosbuvir: alternative in hepatitis C infection?].

Deutsche medizinische Wochenschrift (1946)·2013
Same author

Reforming the EU environment for clinical trials: what are the challenges?

QJM : monthly journal of the Association of Physicians·2013
Same author

[Pathology: pacemaker of advances in medicine].

Deutsche medizinische Wochenschrift (1946)·2013
Same author

Renal grafts from anti-hepatitis B core-positive donors: a quantitative review of the literature.

Transplant infectious disease : an official journal of the Transplantation Society·2012

Area of Science:

  • Molecular Biology
  • Genetics

Context:

  • Recombinant DNA technology facilitates gene cloning and expression.
  • Cloned genes are fundamental to developing human gene therapies.

Purpose:

  • To outline the principles and strategies of human gene therapy.
  • To explain the application of gene therapy in treating genetic disorders.

Summary:

  • Gene therapy employs three main strategies: gene replacement for single-gene defects (Mendelian inheritance), gene augmentation for complex diseases by adding cellular functions, and blocking gene expression for acquired conditions like infections and cancers.
  • Recombinant DNA technology is the cornerstone of these gene therapy approaches, enabling precise genetic modifications.

Impact:

  • Provides a foundational understanding of gene therapy mechanisms.

Related Experiment Videos

  • Highlights the potential of gene therapy in treating a wide range of genetic and acquired diseases.
  • Emphasizes the role of molecular biology tools in advancing therapeutic strategies.