Gene transfer for ocular neovascularization and macular edema

Research output: Contribution to journalArticle

Abstract

Diseases complicated by abnormal growth of vessels or excessive leakage are the most prevalent cause of moderate or severe vision loss in developed countries. Recent progress unraveling the molecular pathogenesis of several of these disease processes has led to new drug therapies that have provided major benefits to patients. However, those treatments often require frequent intraocular injections, and despite monthly injections, some patients have a suboptimal response. Gene transfer of antiangiogenic proteins is an alternative approach that has the potential to provide long-term suppression of neovascularization (NV) and/or excessive vascular leakage in the eye. Studies in animal models of ocular NV have demonstrated impressive results with a number of transgenes, and a clinical trial in patients with advanced neovascular age-related macular degeneration has provided proof-of-concept. Two ongoing clinical trials, one using an adeno-associated viral (AAV) vector to express a vascular endothelial growth factor-binding protein and another using a lentiviral vector to express endostatin and angiostatin, will provide valuable information that should help to inform future trials and provide a foundation on which to build.

Original languageEnglish (US)
Pages (from-to)121-126
Number of pages6
JournalGene Therapy
Volume19
Issue number2
DOIs
StatePublished - Feb 2012

Fingerprint

Macular Edema
Angiostatins
Clinical Trials
Intraocular Injections
Endostatins
Genes
Macular Degeneration
Transgenes
Developed Countries
Vascular Endothelial Growth Factor A
Blood Vessels
Carrier Proteins
Animal Models
Drug Therapy
Injections
Growth
Proteins
Therapeutics

Keywords

  • age-related macular degeneration
  • angiogenesis
  • diabetic retinopathy
  • neovascularization
  • proliferative retinopathies

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

Cite this

Gene transfer for ocular neovascularization and macular edema. / Campochiaro, Peter A.

In: Gene Therapy, Vol. 19, No. 2, 02.2012, p. 121-126.

Research output: Contribution to journalArticle

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