Results 21 to 30 of about 2,056 (145)

Prime Editing for Inherited Retinal Diseases

Frontiers in Genome Editing, 2021
Inherited retinal diseases (IRDs) are chronic, hereditary disorders that lead to progressive degeneration of the retina. Disease etiology originates from a genetic mutation—inherited or de novo—with a majority of IRDs resulting from point mutations ...
Bruna Lopes da Costa, Bruna Lopes da Costa, Sarah R. Levi, Eric Eulau, Yi-Ting Tsai, Yi-Ting Tsai, Peter M. J. Quinn   +6 more
doaj   +1 more source

Frequency of RPE65 Gene Mutation in Patients with Hereditary Retinal Dystrophy

Türk Oftalmoloji Dergisi, 2022
Objectives:Hereditary retinal dystrophies are a rare group of diseases which are heterogeneous in genotype and phenotype and result in total blindness.
Neslihan Sinim Kahraman, Ayşe Öner, Yusuf Özkul, Munis Dündar   +3 more
doaj   +1 more source

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

Frontiers in Immunology, 2022
One of the major goals of in vivo gene transfer is to achieve long-term expression of therapeutic transgenes in terminally differentiated cells. The extensive clinical experience and the recent approval of Luxturna® (Spark Therapeutics, now Roche) and ...
David-Alexandre Gross, David-Alexandre Gross, Novella Tedesco, Novella Tedesco, Christian Leborgne, Christian Leborgne, Giuseppe Ronzitti, Giuseppe Ronzitti   +7 more
doaj   +1 more source

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

, 2020
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
Acosta Acero, Marcy Viviana, Maldonado, Ramiro S, Marcy Acosta-Acero, Ramiro S. Maldonado, Prado, Dominic A, Dominic A. Prado   +5 more
core   +1 more source

Terapia gênica para doenças da retina [PDF]

Revista Brasileira de Oftalmologia
RESUMO Este artigo de atualização tem como objetivo discutir as principais terapias genéticas em estudo para uso nas doenças da retina, principalmente entre as diferentes distrofias hereditárias da retina.
Mariana Matioli da Palma, Mariana Vallim Salles, Juliana Maria Ferraz Sallum   +2 more
doaj   +1 more source

An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

Journal of Market Access & Health Policy, 2021
Background: Gene therapies can treat, prevent, or cure a disease by changing the expression of a person’s genes. They are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare ...
Michał Pochopień, Ewelina Paterak, Emilie Clay, Justyna Janik, Samuel Aballea, Małgorzata Biernikiewicz, Mondher Toumi   +6 more
doaj   +1 more source

Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers

Journal of Ophthalmic & Vision Research, 2020
In this “Perspective”, we discuss ocular gene therapy – the patient's perspective, the various strategies of gene replacement and gene editing, the place of adeno-associated virus vectors, routes of delivery to the eye and the remaining question - “why ...
Geoffrey A. Casey, Kimberly M. Papp, Ian M. MacDonald   +2 more
doaj   +1 more source

Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles. [PDF]

PLoS Pathogens, 2018
The Adeno-associated virus (AAV) gene delivery system is ushering in a new and exciting era in the United States; following the first approved gene therapy (Glybera) in Europe, the FDA has approved a second therapy, Luxturna [1].
J Kennon Smith, Mavis Agbandje-McKenna
doaj   +1 more source

Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey

BMJ Open, 2021
Introduction Voretigene neparvovec-rzyl (Luxturna) was approved by the Australian Therapeutic Goods Administration on 4 August 2020 for the treatment of biallelic mutations in the RPE65 gene, a rare cause of congenital and adult-onset retinal dystrophy ...
, Fred K Chen, Alex W Hewitt, Alan Ma, John Grigg, Keith R Martin, Robyn Jamieson, Heather G Mack, Fleur O’Hare, David Mackey, John De Roach, Alexis Ceecee Britten-Jones, Myra McGuinness, Nicole Tindill, Lauren Ayton, Anai Gonzalez Cordero, Thomas L Edwards, Gladys Ho, Michael Hogden, Anthony Kwan, Tina Lamey, Terri McLaren, Benjamin Nash, Jon Ruddle, Matthew Simunovic, Ingrid Sinnerbrink, Deepa Ajay Taranath, Jen Thompson, Jaclyn White   +28 more
doaj   +1 more source

Cost-Utility Analysis of Luxturna versus the Standard of Care Treatment from a Narrow Danish Societal Perspective [PDF]

, 2020
Introduction: Luxturna is the first gene therapy approved for the treatment of rare disease inherited retinal dystrophy (IRD) with confirmed biallelic RPE65 mutation. The disease impacts patients at a young age and eventually causes blindness.
Morgan, Alexandria Marie, Zheng, Shida
core