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Related Concept Videos

Gene Therapy00:59

Gene Therapy

27.7K
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...
27.7K
Group Therapy01:26

Group Therapy

460
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...
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Gene Flow02:39

Gene Flow

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

Gene Conversion

10.7K
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...
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Gene Families01:57

Gene Families

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Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
10.0K
What is Gene Expression?01:42

What is Gene Expression?

197.1K
Overview
Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
Genetic Information Flows from DNA to RNA to Protein
A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is made up of nucleotides and proteins consist of amino...
197.1K

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Related Experiment Video

Updated: Feb 12, 2026

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
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Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research

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Gene Therapy for Retinal Degeneration.

Rajendra S Apte1

  • 1Washington University School of Medicine, 660 South Euclid Avenue, Box 8096, St. Louis, MO 63110, USA.

Cell
|March 24, 2018
PubMed
Summary

Gene therapy using an adenoviral vector delivers normal RPE65 protein to retinal cells. This treatment restores the visual cycle and vision in patients with inherited retinal degenerations caused by RPE65 gene mutations.

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Inherited retinal degenerations (IRDs) are often caused by biallelic mutations in the RPE65 gene.
  • These mutations disrupt the visual cycle, leading to progressive vision loss.

Purpose of the Study:

  • To develop and evaluate a novel gene therapy for RPE65-associated IRDs.
  • To restore the visual cycle and visual function through gene augmentation.

Main Methods:

  • Surgical delivery of an adenoviral vector carrying the normal human RPE65 gene to retinal cells.
  • Assessment of the visual cycle restoration and visual function recovery.

Main Results:

  • The gene therapy successfully delivered functional RPE65 protein to retinal cells.

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Transpupillary-Guided Trans-Scleral Transplantation of Subretinal Grafts in a Retinal Degeneration Mouse Model
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  • Restoration of the visual cycle and partial recovery of vision were observed.

    • Conclusions:

    • Adenoviral vector-mediated RPE65 gene therapy is a viable approach for treating RPE65-associated IRDs.
    • This 'bench to bedside' therapy offers potential for vision restoration in affected individuals.