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Genes as Medicine

Topic

  • Biochemistry & Molecular Biology
  • Biotechnology
  • Genetics
  • Patterns of Inheritance
  • Genetic Disease

Resource Type

  • Videos
  • Short Films

Level

High School — GeneralHigh School — AP/IBCollege

Duration

00:17:16
Used In
2 BioInteractive Playlists
Saved By
41 Users
Share This
View in Spanish

Description

This film describes the scientific principles and the research efforts involved in the development of a gene therapy for a congenital form of blindness, and how a young patient benefited from this medical breakthrough.

Gene therapy is an approach for treating inherited diseases by delivering corrective versions of genes to patients. Dr. Jean Bennett and Dr. Albert Maguire at the University of Pennsylvania have focused their careers on developing a successful gene therapy for an inherited form of childhood blindness called Leber congenital amaurosis. This film tells the story of their successes and setbacks, while exploring how autosomal recessive conditions are inherited, how scientists use viruses to deliver human genes to cells, and how model organisms are used to test treatments before they are used in patients.

The “Abbreviated Film Guide” provides a short summary of the film, along with key concepts and connections to curriculum standards.

An audio descriptive version of the film is available via our media player.

Key Terms

gene therapy, Leber congenital amaurosis, viral delivery

Primary Literature

Russell, Stephen, Jean Bennett, Jennifer A. Wellman, Daniel C. Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, et al. “Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.” The Lancet 390 (2017): 849–860. https://doi.org/10.1016/S0140-6736(17)31868-8.

Terms of Use

Please see the Terms of Use for information on how this resource can be used.

Accessibility Level (WCAG compliance)

PDF files partially meet criteria. Video files meet criteria. Spanish files meet criteria.

Version History

Date Published 11.22.17
Date Updated 05.14.20

NGSS (2013)

HS-LS1.A, HS-LS3.A, HS-LS3.B

AP Biology (2019)

IST-1, IST-2, IST-3

IB Biology (2016)

3.4, 3.5, B.4

Vision and Change (2009)

CC2, CC3

Materials

HD (M4V) 652 MB
HD (WMV) 544 MB
SD (M4V) 147 MB
SD (WMV) 147 MB
Transcript (PDF) 50 KB
Spanish Dub - Español (MP4) 274 MB
Transcript - Español (PDF) 248 KB
Abbreviated Film Guide (PDF) 731 KB

Use This Resource With

Activity Resource
Activity for Genes as Medicine

Related Science News

Gene therapy for rare form of blindness wins US approval
Officials remove special rules for gene therapy experiments
In a milestone year, gene therapy finds its place in medicine
Doctors try 1st CRISPR editing in the body for blindness

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