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

Progress in human gene therapy

F J Feuerbach1, R G Crystal

  • 1Division of Pulmonary and Critical Care Medicine, New York Hospital-Cornell Medical Center, New York, USA.

Kidney International
|June 1, 1996
PubMed
Summary
This summary is machine-generated.

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

Progress and prospects: gene therapy for performance and appearance enhancement.

Gene therapy·2008
Same author

Assessing disease severity in late infantile neuronal ceroid lipofuscinosis using quantitative MR diffusion-weighted imaging.

AJNR. American journal of neuroradiology·2007
Same author

Neurological deterioration in late infantile neuronal ceroid lipofuscinosis.

Neurology·2007
Same author

AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.

Gene therapy·2005
Same author

VEGF levels in the alveolar compartment do not distinguish between ARDS and hydrostatic pulmonary oedema.

The European respiratory journal·2005
Same author

Effect of adenovirus-mediated expression of Sonic hedgehog gene on hair regrowth in mice with chemotherapy-induced alopecia.

Journal of the National Cancer Institute·2001
Same journal

KDIGO Life Cycle of Guideline Development Series Part 6: Bridging the Gap between Guidelines and Clinical Practice: The KDIGO Approach to Global Implementation and Education in Nephrology.

Kidney international·2026
Same journal

KDIGO Life Cycle of Guideline Development Series Part 5: Guideline updates and a living model for the future.

Kidney international·2026
Same journal

Crystal-storing histiocytosis causing severe acute kidney injury.

Kidney international·2026
Same journal

Peritoneal dialysis in a patient with extensive burn scarring.

Kidney international·2026
Same journal

COPA syndrome unmasked by anti-neutrophil cytoplasmic antibody-positive immune-complex nephritis.

Kidney international·2026
Same journal

Monitoring anti-nephrin antibodies in the management of recurrent diffuse podocytopathy.

Kidney international·2026
See all related articles

Gene therapy offers a promising approach to treating human diseases by transferring genes into cells. While clinical trials show potential and safety, further advancements are needed for widespread therapeutic use.

Area of Science:

  • Molecular Biology
  • Human Pathophysiology
  • Gene Therapy

Background:

  • Recent decades have seen significant progress in understanding disease mechanisms at the molecular level.
  • Advances in gene transfer methods for mammalian cells have fueled interest in gene therapy.
  • Gene therapy aims to treat diseases by introducing therapeutic genes into patient cells.

Purpose of the Study:

  • To review the current state and potential of human gene therapy.
  • To highlight the feasibility and early outcomes of gene transfer in clinical settings.
  • To identify challenges and future directions for gene therapy as a medical treatment.

Main Methods:

  • Review of clinical trials and research on human gene transfer.
  • Analysis of strategies for introducing genes into human cells.

Related Experiment Videos

  • Evaluation of biological responses to transgenes in disease contexts.

    • Main Results:

    • Human gene transfer is technically feasible with various successful strategies.
    • Transgenes can elicit biologically relevant responses for disease treatment.
    • Early clinical trials demonstrate encouraging results with uncommon adverse reactions.
    • Gene transfer provides valuable insights into disease pathophysiology.

    Conclusions:

    • Human gene therapy is a promising technology with demonstrated potential.
    • Overcoming existing obstacles is crucial for establishing gene therapy as a standard treatment.
    • Continued research and development are essential to realize the full benefits of gene therapy for hereditary and acquired diseases.