Results 61 to 70 of about 370,744 (189)

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

Reconceptualizing Pediatric Strabismus as a Condition Rooted in Sensory Processing Disorder: A Novel Case-Based Hypothesis

Children
Background/Objectives: A direct link between sensory processing disorder (SPD) and strabismus has not been systematically investigated, though prior studies suggest sensory modulation may influence visual behaviors. Traditional approaches view strabismus
Mirjana Bjeloš, Ana Ćurić, Mladen Bušić, Katja Rončević, Adrian Elabjer   +4 more
doaj   +1 more source

Fhod3 in zebrafish supports myofibril stability during growth of embryonic skeletal muscle

Developmental Dynamics, EarlyView.
Abstract Background Actin filament organization in cardiomyocytes critically depends on the formin Fhod3, but a role for Fhod3 in skeletal muscle development has not yet been described. Results We demonstrate here that in zebrafish mutated for one of two fhod3 paralog genes, fhod3a, skeletal muscle of the trunk appears normal through 2 days post ...
Aubrie Russell, Tracy Eng, Jeffrey D. Amack, David Pruyne   +3 more
wiley   +1 more source

Development of Refractive Errors—What Can We Learn From Inherited Retinal Dystrophies? [PDF]

, 2017
Purpose It is unknown which retinal cells are involved in the retina-to-sclera signaling cascade causing myopia. As inherited retinal dystrophies (IRD) are characterized by dysfunction of a single retinal cell type and have a high risk of refractive ...
Pierrache, Laurence, Stanley Lambertus, Mies M. van Genderen, Arthur A. Bergen, Wendy A. van Zelst-Stams, F. Nienke Boonstra, Magda Meester-Smoor, Ingeborgh van den Born, L., Virginie J.M. Verhoeven, van Leeuwen, Redmer, Maarten Kamermans, Nathalie M. Bax, Bergen, Arthur A., José Schuil, Clasien J. Oomen, Klevering, B Jeroen, Redmer van Leeuwen, Meester-Smoor, Magda, Oomen, Clasien J, van Schooneveld, Mary J., Mary J. van Schooneveld, Camiel J. Boon, Michelle Hendriks, Boon, Camiel J.F., Laurence Pierrache, Meester-Smoor, Magda A., Boonstra, F Nienke, RD5000 Consortium, Hester Y Kroes, van Genderen, Mies M., Jan Roelof Polling, Caroline C.W. Klaver, B. Jeroen Klevering, van Huet, Ramon A C, Gabriëlle H.S. Buitendijk, Polling, Jan Roelof, Cremers, Frans Pm, Hofman, Albert, Albert Hofman, Klaver, Caroline C W, van Zelst-Stams, Wendy A. G., Ramon A. van Huet, Schuil, José, Magda A. Meester-Smoor, Astrid S. Plomp, L. Ingeborgh van den Born, Verhoeven, Virginie J M, Plomp, Astrid S, Yvonne de Jong-Hesse, Carel B. Hoyng, van den Born, L Ingeborgh, Jan W.R. Pott, Kamermans, Maarten, Buitendijk, Gabriëlle H.S., Bax, Nathalie M., Lambertus, Stanley, Kroes, Hester Y., Pott, Jan W.R., Frans P. Cremers, Reinier O. Schlingemann, Schlingemann, Reinier O, Hoyng, Carel B., Hendriks, Michelle, de Jong-Hesse, Yvonne   +63 more
core   +2 more sources

Inherited metabolic epilepsies–established diseases, new approaches

Epilepsia Open, EarlyView.
Abstract Inherited metabolic epilepsies (IMEs) represent the inherited metabolic disorders (IMDs) in which epilepsy is a prevailing component, often determining other neurodevelopmental outcomes associated with the disorder. The different metabolic pathways affected by individual IMEs are the basis of their rarity and heterogeneity.
Itay Tokatly Latzer, Phillip L. Pearl
wiley   +1 more source

Functional imaging of mitochondria in genetically confirmed retinal dystrophies using flavoprotein fluorescence

, 2022
Whether by indirect oxidative stress or direct genetic defect, various genetic retinal dystrophies involve mitochondrial stress. Mitochondrial flavoprotein fluorescence (FPF), reported as either average signal intensity or variability (heterogeneity ...
Elias I. Traboulsi (7928237), Madhukar Kumar (14132848), Justin C. Muste (14132842), Rishi P. Singh (604020), Matthew W. Russell (14132839), Collin A. Rich (14132851), Kanika Seth (14132845)   +6 more
core   +1 more source

Human biomarker navigator

iMeta, EarlyView.
The Human Biomarker Navigator integrates the disease continuum, biomarker dynamics, cross‐organ biomarker networks, biomarker classification, and technology‐driven paradigms. It maps how biomarkers link multi‐system physiology and pathology across the nervous, respiratory, endocrine, circulatory, immune, digestive, urinary, reproductive, and ...
Meng‐Yao Li, Qian Zhang, Zijie Wang, Zeping Gui, Jing Liu, Jingda Li, Maojun Jiang, Zukang Chang, Qingqing Han, Bendong Yang, Tariq Saeed, Jun Li, Jinghan Jia, Chenghu Song, Tong Shao, Tianhuan Peng, Xueying Zhang, Pingping Guo, Jun Luo, Gangao Yang, Yuan Wan, Gokhan Zengin, Quan Yuan, Lingru Li, Xiaohui Fan, Yang Luo, Lvyun Zhu, Yanfei Zheng, Wenjun Mao, Wenlong Sun   +29 more
wiley   +1 more source

Gene therapy on inherited retinal dystrophies: an update. [PDF]

, 2018
[EN] Inherited retinal dystrophies (IRDs) are a heterogeneous group of disorders characterized by mutations in one of more than 290 identified genes and loci (https://sph.uth.edu/retnet/disease.htm), resulting in the dysfunction or death of ...
Crespo Cruz, Marina Izaskun
core  

Precision Medicine in Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders: An Update on Emerging Treatments

Movement Disorders Clinical Practice, EarlyView.
Abstract Background Neurodegeneration with Brain Iron Accumulation (NBIA) is a heterogeneous group of heritable, mostly recessive, progressive neurodegenerative diseases characterized by iron deposition in the basal ganglia and brainstem. There are no solid global epidemiological data on prevalence and incidence of NBIA subtypes, but registry data and ...
Susanne A. Schneider, Divyani Garg, Vassilena Iankova, Thomas Klopstock   +3 more
wiley   +1 more source

Current Treatment Options for Patients with Inherited Retinal Dystrophies and Ongoing Gene Therapy Clinical Trials [PDF]

Inherited retinal dystrophies affect a great majority of the population, causing millions of people to suffer from these diseases. This systematic review seeks to evaluate current treatment options for patients with leber's congenital amaurosis ...
Henderson, Jaclyn N.
core   +1 more source