Results 61 to 70 of about 169,015 (342)
Advanced Science, EarlyView.Current preclinical studies of AAV‐mediated gene therapy explore different strategies based on the characteristics of inner ear diseases. For genetic hearing loss, approaches include the replacement of a “good gene,” removal of a “bad gene,” or direct correction of mutations through base editing.
Fan Wu +7 more
wiley +1 more source
Orphanet Journal of Rare Diseases, 2019 Purpose Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs.
Ryan S. Paquin +13 more
doaj +1 more source
Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism
Cell Death and Disease, 2023 Facioscapulohumeral muscular dystrophy (FSHD) is among the most common of the muscular dystrophies, affecting nearly 1 in 8000 individuals, and is a cause of profound disability.
Justin Cohen +10 more
doaj +1 more source
The UK myotonic dystrophy patient registry: facilitating and accelerating clinical research [PDF]
, 2017 Myotonic dystrophy type 1 (DM1) is the most frequent muscular dystrophy worldwide with complex, multi-systemic, and progressively worsening symptoms. There is currently no treatment for this inherited disorder and research can be challenging due to the ...
Atalaia, Antonio +18 more
core +4 more sources
Advanced Science, EarlyView.This study investigates the role of group 2 innate lymphoid cells (ILC2) in the choroid plexus (ChP) following traumatic brain injury (TBI). ILC2 accumulation alleviates immune infiltration, preserves hippocampal integrity, and improves sensory‐motor and memory functions.
Shiqi Gao +10 more
wiley +1 more source
CDK inhibitors for muscle stem cell differentiation and self-renewal
Journal of Physical Fitness and Sports Medicine, 2017 Regeneration of muscle is undertaken by muscle stem cell populations named satellite cells which are normally quiescent or at the G0 phase of the cell cycle.
Amrudha Mohan, Atsushi Asakura
doaj +1 more source
Facioscapulohumeral muscular dystrophy
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 2015 Facioscapulohumeral muscular dystrophy (FSHD) is characterized by a typical and asymmetric pattern of muscle involvement and disease progression. Two forms of FSHD, FSHD1 and FSHD2, have been identified displaying identical clinical phenotype but different genetic and epigenetic basis.
Sacconi, Sabrina +2 more
openaire +5 more sources
Muscular dystrophy meets protein biochemistry, the mother of invention [PDF]
, 2017 Muscular dystrophies result from a defect in the linkage between the muscle fiber cytoskeleton and the basement membrane (BM). Congenital muscular dystrophy type MDC1A is caused by mutations in laminin α2 that either reduce its expression or impair its ...
Jeffrey H. Miner +3 more
core +2 more sources
Recent Advances in mRNA Delivery Systems for Cancer Therapy
Advanced Science, EarlyView.This review systematically investigates the applications of mRNA therapy in cancer treatment, with particular emphasis on nonviral delivery systems, targeting strategies, stimulus‐responsive systems, and local delivery methods. Concluding with a meticulous evaluation, the review sheds light on the prevailing challenges while illuminating promising ...
Zheng Zhang +9 more
wiley +1 more source