Results 111 to 120 of about 2,056 (145)

Visualising treatment effects in low-vision settings: proven and potential endpoints for clinical trials of inherited retinal disease therapies. [PDF]

Gene Ther
Thirunavukarasu AJ, Raji S, Cehajic Kapetanovic J.   +2 more
europepmc   +1 more source

Gene therapy trial lights the way for patients with Leber congenital amaurosis 1. [PDF]

Mol Ther
Chan YK.
europepmc   +1 more source

Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization. [PDF]

Int J Mol Sci
Tsaregorodtseva TS, Radyukhina MA, Ayupova AI, Solovyeva VV, Sufianov AA, Sufianova GZ, Rizvanov AA.   +6 more
europepmc   +1 more source

The origins of Novo Nordisk and Novartis products: piloting a framework to identify the public contributions. [PDF]

J Pharm Policy Pract
Fabian D, Sehic O, Wild C.
europepmc   +1 more source

A hierarchical and configurational analysis of Health Technology Assessment outcomes for cell and gene therapies. [PDF]

Front Pharmacol
Almalki ZS, Alshammari RM, Dahduli NA, Nagi MA, Juweria S, Alhamdani MM, Alzahrani YM, Almazrou SH, Ahmed NJ, Alahmari AK, Alshlowi AA.   +10 more
europepmc   +1 more source

Disparities in access to gene therapy in the European Union: ethical and regulatory challenges. [PDF]

J Law Biosci
Reckelbus M, Mohan R, Locquet P, Van Steijvoort E, Huys I, Borry P.   +5 more
europepmc   +1 more source

Streamlined synthetic regulatory cassette for efficient and photoreceptor-enriched retinal gene expression. [PDF]

Mol Ther Nucleic Acids
Kwon JE, Kim YJ, Kim SH, Kwon OS, Jang HK, Choi YM, Na HJ, Han N, Chun Y, Han JW, Park TK, Chung KS.   +11 more
europepmc   +1 more source

Should patients with pre-existing anti-AAV antibodies be excluded from receiving intrathecally delivered AAV vectors? [PDF]

Mol Ther Methods Clin Dev
Gray SJ.
europepmc   +1 more source

Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates. [PDF]

Mol Ther Adv
Sourd C, Quinn J, John MC, Martinez-Fernandez de la Camara C, Wickramasinghe LC, Attar M, Shamsnajafabadi H, Salman A, Cowley SA, Dendrou CA, MacLaren RE, Cehajic-Kapetanovic J, Xue K.   +12 more
europepmc   +1 more source

Advances and Challenges in Gene Therapy for Inherited Retinal Dystrophies: A Comprehensive Review. [PDF]

Cureus
Jain R, Daigavane S.
europepmc   +1 more source