Results 161 to 170 of about 5,172,195 (247)

Gene Therapy for Inherited Retinal Disease: Current Strategies, Personalized Medicine, and Future Implications-A Comprehensive Review. [PDF]

J Pers Med
Butt FR, Dhivagaran T, Li B, Ashamalla M, Tao BK, Balas M, Pereira A, Yan P, Arjmand P.   +8 more
europepmc   +1 more source

Monitoring pharmacodynamic and molecular drug targets in liquid biopsy: Exploratory study in liver cancer with modelling of EGFR Receptor engagement

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Liquid biopsy is minimally invasive (compared with tissue biopsy) and has previously been used to generate systems data regarding drug elimination via hepatic enzymes and transporters. This study extends quantitative assessment of systems parameters in liquid biopsy to pharmacodynamic (PD) and disease markers relevant to cancer development
Zubida M. Al‐Majdoub, Armin Sepp, Amin Rostami‐Hodjegan, Brahim Achour   +3 more
wiley   +1 more source

AAV-delivered engineered suppressor tRNA rescues visual function in mice with an inherited retinal disease. [PDF]

Nat Commun
Ren C, Song L, Hu M, Jin X, Liang L, Liu F, Su J, Fu J, Yang Y, Liu M, Xu Q, Wu X, Luo M, Ye Q, Li Q, An Y, Li M, She K, Wei Y, Lu F, Yang Y.   +20 more
europepmc   +1 more source

Machine Learning Models of Phase Contrast Images Predict Efficiency of Human Pluripotent Stem Cell Differentiation to Cardiomyocytes

Biotechnology and Bioengineering, EarlyView.
Human pluripotent stem cells were differentiated to cTnT+ cardiomyocytes and imaged throughout the process using phase contrast. We predicted differentiation efficiency (cTnT+ area) from phase contrast and compared model performance with a live‐cell cTnT‐GFP reporter.
Austin K. Feeney, Yaniv Ovadia, Aaron D. Simmons, Christian Elabd, Claire J. Peplinski, Frank Li, Sean P. Palecek   +6 more
wiley   +1 more source

One down but many more to go: the state of gene therapy for inherited retinal disease. [PDF]

Regen Med
Tan TE, Sun CZY, Poh SSJ, Lim J, Teo JXJ, Dey S, Chua ZWX, Guo J, Wang Z, Tay HG, Fenner BJ.   +10 more
europepmc   +1 more source

Human retina-in-a-dish: Unlocking the potential to study mechanisms of inherited retinal disease. [PDF]

Mol Ther Methods Clin Dev, 2023
Yee T, Wert KJ.
europepmc   +1 more source

What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?

Biotechnology and Bioengineering, EarlyView.
Several host cell types have been used to produce rAAVs to date. Cell line‐specific traits that are beneficial in the context of rAAV manufacturing are reviewed here, with the goal of developing a consensus on the ‘ideal’ characteristics that an rAAV production host should possess (created with Biorender.com).
James Conheady, Nicholas Donohue, Alexandra Bogdanovic, Sharon Davin, Brian Glennon, Niall Barron, C. Mark Smales   +6 more
wiley   +1 more source

Charles Bonnet syndrome in adults with inherited retinal disease: prevalence and patient perspectives. [PDF]

BMJ Open Ophthalmol
Weatherby TJM, Boyle M, Madhusudhan S.
europepmc   +1 more source

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

Biotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga, Udisha Singh, Sachin Dattram Pawar   +2 more
wiley   +1 more source

Clinical Research for Inherited Retinal Disease Related Pediatric Blindness: A Preliminary Descriptive Analysis Based on ClinicalTrials.gov. [PDF]

J Multidiscip Healthc
Ashour AM, Harbi MH, Alshehri FS, Wali SM, Aldurdunji MM, Alorfi NM.   +5 more
europepmc   +1 more source