Promises and challenges of genetic therapy for blindness

doi:10.1016/S0140-6736(09)61869-9

The Lancet

Volume 374, Issue 9701, 7–13 November 2009, Pages 1569-1570

https://doi.org/10.1016/S0140-6736(09)61869-9 Get rights and content

References (12)

NS Dejneka et al.
In utero gene therapy rescues vision in a murine model of congenital blindness
Mol Ther
(2004)
GM Acland et al.
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
Mol Ther
(2005)
AI den Hollander et al.
Leber congenital amaurosis: genes, proteins and disease mechanisms
Prog Retin Eye Res
(2008)
JWB Bainbridge et al.
Effect of gene therapy on visual function in Leber's congenital amaurosis
N Engl J Med
(2008)
AM Maguire et al.
Safety and efficacy of gene transfer for Leber's congenital amaurosis
N Engl J Med
(2008)
AV Cideciyan et al.
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
Proc Natl Acad Sci USA
(2008)

There are more references available in the full text version of this article.

Cited by (8)

Gene therapy as a new treatment option for inherited monogenic diseases
2014, European Journal of Internal Medicine
Citation Excerpt :
Earlier intervention, when there is less photoreceptor damage, resulted in gains in ambulatory vision. This is an important observation for diseases affecting tissues with limited regenerative capacities [60]. Most recently, a study demonstrated that the peripheral infusion of an AAV8 vector expressing a codon-optimized human factor IX transgene in six patients with severe hemophilia B resulted in long term expression of factor IX of up to 11% of normal levels with an acceptable safety profile [61].
Gene therapy, replacing a defective gene by a functional copy, has been in development for more than 40 years. Initial efforts involved engineering viral vectors to deliver genes to the appropriate cells. Early successes in severe combined immunodeficiency (SCID) were later derailed by safety issues including host reaction to the vector and gene insertion near promoters that favored secondary leukemia.
Systematic review of the literature using PubMed.gov with key word gene therapy from 1972 to March 2013. Google search with key word gene therapy.
Despite early setbacks, progresses for monogenic diseases continued unabated. Patients with SCIDs have been cured and the first gene therapy has been approved for lipoprotein lipase deficiency. Many clinical research studies are ongoing as part of systematic clinical development program with a view to have more gene therapies approved.
Our review highlights progresses and questions that remain to be answered to make gene therapy an integral part of our therapeutic arsenal.
Bat Biology, Genomes, and the Bat1K Project: To Generate Chromosome-Level Genomes for All Living Bat Species
2018, Annual Review of Animal Biosciences
DAPT mediates atoh1 expression to induce hair cell-like cells
2016, American Journal of Translational Research
NGS-based clinical diagnosis of genetically heterogeneous disorders
2013, Next Generation Sequencing: Translation to Clinical Diagnostics
Childhood eye disorders and visual impairment
2012, Handbook of Children with Special Health Care Needs
High-resolution homozygosity mapping is a powerful tool to detect novel mutations causative of autosomal recessive RP in the Dutch population
2011, Investigative Ophthalmology and Visual Science

View all citing articles on Scopus

View full text

Article preview

The Lancet

References (12)

In utero gene therapy rescues vision in a murine model of congenital blindness

Mol Ther

Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness

Mol Ther

Leber congenital amaurosis: genes, proteins and disease mechanisms

Prog Retin Eye Res

Effect of gene therapy on visual function in Leber's congenital amaurosis

N Engl J Med

Safety and efficacy of gene transfer for Leber's congenital amaurosis

N Engl J Med

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Proc Natl Acad Sci USA

Cited by (8)

Gene therapy as a new treatment option for inherited monogenic diseases

Bat Biology, Genomes, and the Bat1K Project: To Generate Chromosome-Level Genomes for All Living Bat Species

DAPT mediates atoh1 expression to induce hair cell-like cells

NGS-based clinical diagnosis of genetically heterogeneous disorders

Childhood eye disorders and visual impairment

High-resolution homozygosity mapping is a powerful tool to detect novel mutations causative of autosomal recessive RP in the Dutch population

CommentPromises and challenges of genetic therapy for blindness

Mol Ther

Mol Ther

Prog Retin Eye Res

Effect of gene therapy on visual function in Leber's congenital amaurosis

N Engl J Med

Safety and efficacy of gene transfer for Leber's congenital amaurosis

N Engl J Med

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Proc Natl Acad Sci USA

Comment
Promises and challenges of genetic therapy for blindness