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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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Stem Cell Therapy for Tissue Regeneration01:21

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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
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Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells
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Corneal Regeneration Using Gene Therapy Approaches.

Subhradeep Sarkar1,2, Priyalakshmi Panikker1, Sharon D'Souza3

  • 1GROW Research Laboratory, Narayana Nethralaya Foundation, Bangalore 560099, Karnataka, India.

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|May 13, 2023
PubMed
Summary

Gene therapy offers a promising alternative to corneal transplantation for treating eye diseases and reducing blindness. This approach leverages the cornea's unique properties for effective gene delivery, addressing challenges like graft rejection and donor shortages.

Keywords:
adeno-associated viruscorneal dystrophiescorneal neovascularizationgene therapyregenerative medicineviral vectors

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Area of Science:

  • Ophthalmology
  • Regenerative Medicine
  • Gene Therapy

Background:

  • Corneal transplantation is a key treatment for corneal diseases but faces challenges like graft rejection, donor scarcity, and complications.
  • Corneal defects are a leading cause of global blindness, necessitating innovative therapeutic strategies.
  • Gene therapy presents a potential solution to overcome the limitations of current corneal treatments.

Purpose of the Study:

  • To review recent advancements in corneal regeneration using gene therapy.
  • To explore strategies for developing gene-based therapies for corneal diseases.
  • To discuss the challenges, potential, and translational aspects of gene therapy in ophthalmology.

Main Methods:

  • Review of current literature on gene therapy applications in corneal regeneration.
  • Analysis of different gene therapy vectors, with a focus on recombinant adeno-associated virus (AAV).
  • Discussion of in vivo and ex vivo gene therapy approaches for corneal defects.

Main Results:

  • The cornea's immune-privileged, avascular, and transparent nature makes it suitable for gene therapy.
  • Established surgical techniques facilitate gene delivery for corneal conditions.
  • Recombinant AAV vectors show potential for targeted therapeutic delivery in the cornea.

Conclusions:

  • Gene therapy holds significant potential to address challenges in corneal transplantation and reduce blindness.
  • Further research and development are needed to translate gene therapy into effective clinical treatments for corneal diseases.
  • Targeted gene delivery strategies, particularly using AAV, are crucial for advancing corneal regenerative medicine.