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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Types of Genetic Transfer Between Organisms02:18

Types of Genetic Transfer Between Organisms

Genetic transfer occurs when genetic information is passed from one organism to another. It occurs via two mechanisms: vertical gene transfer and horizontal gene transfer. Vertical gene transfer occurs when genetic information is transferred from one generation to the next, which happens much more frequently than horizontal gene transfer. Both sexual and asexual reproduction are forms of vertical gene transfer, where one or more organisms pass some or all of their genome onto their progeny.
Types of Genetic Transfer Between Organisms02:18

Types of Genetic Transfer Between Organisms

Genetic transfer occurs when genetic information is passed from one organism to another. It occurs via two mechanisms: vertical gene transfer and horizontal gene transfer. Vertical gene transfer occurs when genetic information is transferred from one generation to the next, which happens much more frequently than horizontal gene transfer. Both sexual and asexual reproduction are forms of vertical gene transfer, where one or more organisms pass some or all of their genome onto their progeny.
Leaky Scanning02:28

Leaky Scanning

During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA.  Marilyn Kozak discovered that the sequence RCCAUGG (where R stands for...
Transgenic Organisms00:53

Transgenic Organisms

Overview

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An Efficient In Vitro Transposition Method by a Transcriptionally Regulated Sleeping Beauty System Packaged into an Integration Defective Lentiviral Vector
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Translating gene transfer: a stalled effort.

Alexandra J Greenberg1, Jennifer McCormick, Carmen J Tapia

  • 1Mayo Clinic College of Medicine, Center for Translational Science Activities, Rochester, Minnesota, USA. greenberg.alexandra@mayo.edu

Clinical and Translational Science
|September 3, 2011
PubMed
Summary
This summary is machine-generated.

Gene transfer research, starting in the 1970s, faces significant challenges moving from lab to clinic. Examining its history highlights crucial lessons for future gene therapy development and public engagement.

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Area of Science:

  • Biotechnology and Translational Medicine
  • Molecular Biology and Genetics
  • Medical Ethics and Public Health

Background:

  • Gene transfer technology emerged in the early 1970s, with significant interest in the 1990s.
  • Despite decades of research, a standardized clinical application of gene therapy has not been established.
  • The translational journey of gene transfer has encountered numerous obstacles and setbacks.

Purpose of the Study:

  • To examine the historical progress of gene transfer from laboratory research to clinical application.
  • To identify key challenges, successes, and lessons learned from the gene transfer translational pathway.
  • To underscore the importance of ethical considerations, safety protocols, and public interaction in advancing gene therapy.

Main Methods:

  • Historical review and analysis of gene transfer research and development.
  • Examination of documented successes and failures in the translation of gene transfer technologies.
  • Case study approach to understand the evolution of gene therapy's clinical integration.

Main Results:

  • The clinical translation of gene transfer has been slower than anticipated due to various barriers.
  • Past research and development provide valuable insights into navigating ethical and safety concerns.
  • Effective scientist-public engagement is critical for the successful advancement of gene therapy.

Conclusions:

  • The history of gene transfer offers a crucial learning model for developing future gene therapies.
  • Addressing ethical, safety, and public perception issues is paramount for clinical gene therapy success.
  • Continued examination of the translational journey is essential for optimizing gene therapy's path to widespread clinical use.