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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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Targeted Cancer Therapies

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
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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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Related Experiment Video

Updated: Feb 10, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
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Prospects for retinal cone-targeted gene therapy.

John J Alexander1, William W Hauswirth

  • 1Department of Pathology, Immunology, and Laboratory Medicine, University of Florida College of Medicine, Gainesville, Florida, USA.

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|July 4, 2008
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Summary

Gene therapy successfully restored vision in a mouse model of achromatopsia. This approach targets cone photoreceptors, offering hope for treating blinding cone diseases.

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Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Cone photoreceptor diseases cause severe vision loss and eventual blindness.
  • Achromatopsia is a genetic cone disorder rendering cones nonfunctional.

Purpose of the Study:

  • To evaluate gene therapy targeting cone photoreceptors for achromatopsia.
  • To assess the efficacy of adeno-associated virus (AAV) vectors for cone-specific gene delivery.

Main Methods:

  • Utilized the Gnat2(cpfl3) mouse model of achromatopsia.
  • Employed AAV vectors with a cone-specific promoter for gene delivery.
  • Administered a single subretinal injection of the therapeutic vector.

Main Results:

  • Demonstrated recovery of normal cone function in treated mice.
  • Showed restoration of visual acuity following gene therapy.
  • Confirmed successful delivery of wild-type Gnat2 protein to cones.

Conclusions:

  • Gene therapy targeting cones is a viable strategy for achromatopsia.
  • AAV vectors efficiently transduce cones for therapeutic gene delivery.
  • Further investigation in animal models supports potential clinical trials for cone diseases.