Results 251 to 260 of about 131,610 (332)

Balancing Promise and Peril: Hemophilia Gene Therapy Insights. [PDF]

IUBMB Life
Akula S, Borroni E, Cottonaro A, Follenzi A, Merlin S.   +4 more
europepmc   +1 more source

AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice

, 2017
Cristina Mallol, Estefanía Casana, Verónica Jiménez, Alba Casellas, Virginia Haurigot, Claudia Jambrina, Victor Sacristan, Meritxell Morró, Judith Agudo, Laia Vilà, Fátima Bosch   +10 more
openalex   +1 more source

A Nonviral Neo‐Nucleocapsid for Cell‐Specific RNA Delivery Developed by Pseudo‐Cyclic Peptide Grafting and Directed Evolution

Angewandte Chemie, EarlyView.
Nonviral neo‐nucleocapsids developed from a repurposed bacterial protein cage, and macrocyclic peptide pharmacophores via protein‐protein ligation, bacteria‐based directed evolution, lasso‐grafting, and mammalian cell‐based directed evolution enabled cell specific RNA delivery.
Daiki Kanayama, Naohiro Terasaka, Kazuki Kato, Sae Okazaki, Hiroaki Suga   +4 more
wiley   +2 more sources

Lyophilization of Adeno-Associated Virus Serotypes for Storage and Global Distribution. [PDF]

Biomedicines
McGlinch EB, Mudrick HE, Evans CH, Barry MA.   +3 more
europepmc   +1 more source

Co‐Opting MBNL‐Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy

Annals of Neurology, EarlyView.
Objective Myotonic dystrophy type 1 (DM1) is a highly variable, multisystemic genetic disorder caused by a CTG repeat expansion in the 3′ untranslated region of DMPK. Toxicity is exerted by repeat‐containing DMPK transcripts that sequester muscleblind‐like (MBNL) proteins and lead to deleterious yet predictable changes in alternative splicing.
Samuel T. Carrell, Ellie M. Carrell, Ryan Giovenco, Beverly L. Davidson   +3 more
wiley   +1 more source

Combine mitochondrial-targeted gene therapy and chemotherapy to treat triple-negative breast cancer. [PDF]

J Exp Clin Cancer Res
Varadkar T, Zhou ZZ, Zhang J, Sarkar A, Du Z, Chowdhury S, Lim H, Zhou L, Liu XM.   +8 more
europepmc   +1 more source

Skeletal Muscle Biomarkers of Amyotrophic Lateral Sclerosis: A Large‐Scale, Multi‐Cohort Proteomic Study

Annals of Neurology, EarlyView.
Objective Biomarkers with clear contexts of use are important tools for amyotrophic lateral sclerosis (ALS) therapy development. Understanding their longitudinal trajectory in the untreated state is key to their use as potential markers of pharmacodynamic response.
Oleksandr Dergai, Joanne Wuu, Magdalena Koziczak‐Holbro, Andrea Malaspina, Volkan Granit, Jessica P. Hernandez, Anne Cooley, Ruchika Sachdev, Lili Yu, Michael Bidinosti, Ludivine Flotte, Mark Nash, Lori L. Jennings, James D. Berry, Lucie I. Bruijn, Sophie Brachat, Michael Benatar   +16 more
wiley   +1 more source

Biomarkers Detecting the Activity of ANCA-Associated Vasculitis: A Systematic Literature Review. [PDF]

Int J Nephrol
Baas L, Krol RM, Hagen EC, Spierings J, Teng YKO, Koelman CA, Remmelts HHF.   +6 more
europepmc   +1 more source

Decreased scleral Wnt5a^hi fibroblasts exacerbate myopia progression by disrupting extracellular matrix homeostasis in mice. [PDF]

Nat Commun
Zhu H, Chen W, Ling X, Jiao S, Yu L, Liu H, Ding M, Zhang F, Zhou Y, Pan Y, Zhou Z, Qu J, Zhao F, Zhao F, Zhou X.   +14 more
europepmc   +1 more source

Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation [PDF]

Marti Cabanes‐Creus, Sophia H.Y. Liao, Renina Gale Navarro, Maddison Knight, Deborah Nazareth, Ngee‐Soon Lau, Mark Ly, Erhua Zhu, Ramon Roca-Pinilla, Ricardo Bugallo Delgado, Ana F. Vicente, Grober Baltazar, Adrian Westhaus, Jessica Merjane, Michael Crawford, Geoffrey W. McCaughan, Carmen Unzu, Gloria González‐Aseguinolaza, Ian E. Alexander, Carlo Pulitanò, Leszek Lisowski   +20 more
openalex   +1 more source