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Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease.

Publication ,  Journal Article
Prado, DA; Acosta-Acero, M; Maldonado, RS
Published in: Curr Opin Ophthalmol
May 2020

PURPOSE OF REVIEW: Gene therapy offers, for the first time, the possibility to cure diseases such as retinitis pigmentosa. The positive outcomes that led to the U.S. Food and Drug Administration (FDA) approval of Luxturna to treat Leber congenital amaurosis caused by RPE65 mutations created an optimistic atmosphere in the research, clinical and patient community. Despite this first success, we must understand that this is not a 'one treatment for all'. This review aims to explain the basic concepts of gene therapy and how they translate in different approaches that are utilized in ongoing clinical trials here reviewed. RECENT FINDINGS: In 2017, the FDA approved the first gene therapy treatment. In parallel, other approaches have gained attention. Different delivery methods (adeno-associated virus, lentivirus), injection sites (subretinal, intravitreal, suprachoroidal) and methodologies (gene replacement, silencing, editing) are currently being tested. SUMMARY: Gene therapy is an evolving field in medicine and ophthalmology. Its success and application depends on several factors that are specific to the disease to treat. For now, we know it's a relatively safe approach and we look forward to the continued advancements of current ongoing clinical trials.

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Published In

Curr Opin Ophthalmol

DOI

EISSN

1531-7021

Publication Date

May 2020

Volume

31

Issue

3

Start / End Page

147 / 154

Location

United States

Related Subject Headings

  • Retinal Degeneration
  • Ophthalmology & Optometry
  • Lentivirus
  • Humans
  • Genetic Vectors
  • Genetic Therapy
  • Gene Silencing
  • Gene Editing
  • Dependovirus
  • 3212 Ophthalmology and optometry
 

Citation

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Prado, D. A., Acosta-Acero, M., & Maldonado, R. S. (2020). Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease. Curr Opin Ophthalmol, 31(3), 147–154. https://doi.org/10.1097/ICU.0000000000000660
Prado, Dominic A., Marcy Acosta-Acero, and Ramiro S. Maldonado. “Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease.Curr Opin Ophthalmol 31, no. 3 (May 2020): 147–54. https://doi.org/10.1097/ICU.0000000000000660.
Prado DA, Acosta-Acero M, Maldonado RS. Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease. Curr Opin Ophthalmol. 2020 May;31(3):147–54.
Prado, Dominic A., et al. “Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease.Curr Opin Ophthalmol, vol. 31, no. 3, May 2020, pp. 147–54. Pubmed, doi:10.1097/ICU.0000000000000660.
Prado DA, Acosta-Acero M, Maldonado RS. Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease. Curr Opin Ophthalmol. 2020 May;31(3):147–154.

Published In

Curr Opin Ophthalmol

DOI

EISSN

1531-7021

Publication Date

May 2020

Volume

31

Issue

3

Start / End Page

147 / 154

Location

United States

Related Subject Headings

  • Retinal Degeneration
  • Ophthalmology & Optometry
  • Lentivirus
  • Humans
  • Genetic Vectors
  • Genetic Therapy
  • Gene Silencing
  • Gene Editing
  • Dependovirus
  • 3212 Ophthalmology and optometry