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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...
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Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy01:30

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Various diagnostic tests are employed in the diagnostic process for Inflammatory Bowel Disease (IBD), particularly to differentiate between Crohn's disease and ulcerative colitis.
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...
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Group Therapy01:26

Group Therapy

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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 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...
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Gene Conversion02:08

Gene Conversion

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

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Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

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Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.

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Related Experiment Video

Updated: Feb 13, 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 in optic nerve disease.

Adam DeBusk1, Mark L Moster2

  • 1Department of Ophthalmology.

Current Opinion in Ophthalmology
|March 15, 2018
PubMed
Summary

Gene therapy offers promising treatments for optic nerve diseases, promoting axon regeneration and neuroprotection. Clinical trials show safety and feasibility, with animal studies demonstrating successful RGC regrowth and survival.

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Area of Science:

  • Ophthalmology
  • Neuroscience
  • Regenerative Medicine

Background:

  • Optic nerve damage leads to irreversible vision loss due to retinal ganglion cell (RGC) apoptosis.
  • Gene therapy presents a novel approach to address optic nerve disorders by targeting genetic mutations and regeneration pathways.

Purpose of the Study:

  • To review recent advancements in gene therapy and gene modification for optic nerve diseases.
  • To explore the potential of these therapies in promoting axon regeneration, neuroprotection, and visual function recovery.

Main Methods:

  • Review of current literature on gene therapy applications in optic nerve regeneration and neuroprotection.
  • Analysis of findings from clinical trials and animal studies investigating gene modification strategies.

Main Results:

  • Gene therapy has shown potential in replacing gene mutations and modulating regeneration pathways for optic nerve repair.
  • Clinical trials for Leber's Hereditary Optic Neuropathy indicate safety and feasibility.
  • Animal studies demonstrate varying degrees of RGC axon regrowth, target reinnervation, and enhanced RGC survival post-injury.

Conclusions:

  • Gene therapy holds promise for treating optic nerve damage, offering neuroprotection and promoting axon regeneration.
  • Further research is necessary to assess long-term efficacy and safety, including the implications of manipulating tumor suppressor genes and oncogenes.