Results 161 to 170 of about 71,442 (257)

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Science, 2016
Christopher E. Nelson, Chady H. Hakim, David G. Ousterout, P. Thakore, E. A. Moreb, R. Rivera, S. Madhavan, Xiufang Pan, F. Ran, Winston X. Yan, A. Asokan, Feng Zhang, D. Duan, C. Gersbach   +13 more
semanticscholar   +1 more source

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

Small Methods, Volume 10, Issue 2, 22 January 2026.
The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source

Impending A New Epoch In Duchenne Muscular Dystrophy (DMD) Treatment

, 2017
Pramod Khatri
openalex   +1 more source

Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease [PDF]

, 2020
Ariana P. Mullin, Diane Corey, Emily C. Turner, Richard Liwski, Daniel Olson, Jackson Burton, Sudhir Sivakumaran, Lynn D. Hudson, Klaus Romero, Diane Stephenson, Jane Larkindale   +10 more
openalex   +1 more source

Can You See Us Play? Observing Inclusive Outdoor Play Behaviour Among Children With and Without Disabilities: A Mixed Methods Study

Child: Care, Health and Development, Volume 52, Issue 1, January 2026.
ABSTRACT Background Outdoor play is essential for children's development. However, children with disabilities face barriers in outdoor play. In the Netherlands, there are limited opportunities for children with and without disabilities to play together.
R. Q. Beekhuizen, M. A. T. Bloemen, E. A. M. Bolster, K. Visser, R. van der Lugt, M. Bassa, N. L. Henry, E. M. W. Kotte, M. van Hartingsveldt, J. W. Gorter   +9 more
wiley   +1 more source

Climate Change Will Resize and Reshape Plant–Hummingbird Networks in the Atlantic Forest

Diversity and Distributions, Volume 32, Issue 1, January 2026.
ABSTRACT Aim To assess how climate change could reshape plant–hummingbird interaction networks in the Atlantic Forest by predicting shifts in species co‐occurrence and evaluating changes in network structure. Location Atlantic Forest, South America.
Alejandro Restrepo‐González, Thais B. Zanata, Fernanda T. Brum, Silvana Buzato, Catherine H. Graham, Joice Iamara‐Nogueira, Isabela G. Varassin   +6 more
wiley   +1 more source

Longitudinal trajectories of muscle impairments in growing boys with Duchenne muscular dystrophy [PDF]

Ines Vandekerckhove, Marleen Van den Hauwe, Tijl Dewit, Geert Molenberghs, Nathalie Goemans, Liesbeth De Waele, Anja Van Campenhout, Friedl De Groote, Kaat Desloovere   +8 more
openalex   +1 more source

Volitional exercise elicits physiological and molecular improvements in the severe D2.mdx mouse model of Duchenne muscular dystrophy

The Journal of Physiology, Volume 604, Issue 2, Page 849-867, 15 January 2026.
Abstract figure legend This study investigated the effects of volitional exercise on muscle health in the more severe D2.mdx model of Duchenne muscular dystrophy (DMD). We showed that 8–10 weeks of a relatively high volume of voluntary wheel running (VWR) in D2.mdx animals augmented select muscle mass and normalized ex vivo muscle force compared to ...
Stephanie R. Mattina, Sean Y. Ng, Andrew I. Mikhail, Derek W. Stouth, Cora E. Jornacion, Irena A. Rebalka, Thomas J. Hawke, Vladimir Ljubicic   +7 more
wiley   +1 more source

RNA‐Seq of Cultured Peripheral Blood Lymphocytes Improves Identification of Cryptic Splicing Defects in Rare Disease Diagnostics

Human Mutation, Volume 2026, Issue 1, 2026.
Accurate identification of the genetic determinants of rare diseases is essential for effective recurrence‐risk management and informed reproductive decision‐making. Although whole‐exome sequencing (WES) and whole‐genome sequencing (WGS) have significantly improved diagnostic capabilities, a subset of affected families still receives no definitive ...
Jinlin Ren, Congling Dai, Fei Meng, Pan Zhang, Chunbo Xie, Wenjuan Xiao, Wenbin He, Shimin Yuan, Xiurong Li, Qianjun Zhang, Weiling Tang, Liang Hu, Zixu Chen, Guangxiu Lu, Juan Du, Sicong Zeng, Ge Lin, Stuart Scott   +17 more
wiley   +1 more source

The clinical and molecular genetic approach to Duchenne and Becker muscular dystrophy: an updated protocol. [PDF]

, 1997
A. J. van Essen, Alexander L.J. Kneppers, Annemarie H. van der Hout, Hans Scheffer, Ieke B. Ginjaar, Leo P. ten Kate, G J van Ommen, Charles H.C.M. Buys, Egbert Bakker   +8 more
openalex   +1 more source