Results 211 to 220 of about 172,174 (293)

Parental Experiences with Newborn Screening and Gene Replacement Therapy for Spinal Muscular Atrophy. [PDF]

J Neuromuscul Dis
Meyer AP, Connolly AM, Vannatta K, Hacker N, Hatfield A, Decipeda A, Parker P, Willoughby A, Waldrop MA.   +8 more
europepmc   +1 more source

Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. [PDF]

Signal Transduct Target Ther
Wang J, Zhang J, Yu S, Li H, Chen S, Luo J, Wang H, Guan Y, Zhang H, Yin S, Wang H, Li H, Liu J, Zhu J, Yang Q, Sha Y, Zhang C, Yang Y, Yang X, Zhang X, Zhao X, Wang L, Yang L, Wei W.   +23 more
europepmc   +1 more source

Incorporation of Selenium into Sol–Gel‐Derived Bioactive Glass: Influence on Glass Structure, Bioactivity, and its Selective Cytotoxicity

Advanced Engineering Materials, EarlyView.
Selenium was incorporated into a sol–gel‐derived bioactive glass to enable sustained therapeutic ion release. The selenium‐containing glass preserved bioactivity while selectively inducing cytotoxicity in osteosarcoma cells and maintaining osteoblastic viability.
Breno Rocha Barrioni, Thalita Marcolan Valverde, Talita Martins, Guilherme Mattos Jardim Costa, Alfredo Miranda de Goes, Marcello Rosa Dumont, Eduardo Henrique Martins Nunes, Marivalda de Magalhães Pereira   +7 more
wiley   +1 more source

The application of enzyme replacement therapy in vitro and in a mouse model in aspartylglycosaminuria [PDF]

Dunder, Ulla
core  

Designing Polymer Nanocomposites for X‐Ray Shielding: Mechanisms, Architectures, and Scalable Processing

Advanced Engineering Materials, EarlyView.
This review highlights advances in lightweight, lead‐free polymer nanocomposites for diagnostic X‐ray shielding. By linking filler chemistry, dispersion, architecture, and photon interaction mechanisms, it establishes structure–performance relationships guiding material design.
Aklilu G. Messele, Hossein Ipakchi, Tizazu H. Mekonnen   +2 more
wiley   +1 more source

Microenvironmental Reprogramming by 3D Anisotropic Cardiac Extracellular Matrix Induces Nuclear Remodeling and Epigenetic Maturation of Chemically Induced Cardiomyocytes

Advanced Functional Materials, EarlyView.
A 3D anisotropic hydrogel derived from heart extracellular matrix guides cytoskeletal alignment and nuclear remodeling in reprogrammed cardiomyocyte‐like cells. This study reveals how matrix alignment modulates nuclear envelope dynamics and chromatin state, triggering transcriptional and functional maturation.
Seung Ju Seo, Mi Jeong Lee, Hyun Wook Kang, Seonhee Byeon, Soo‐Kyoung Choi, Nakwon Choi, Seung‐Woo Cho, Yoonhee Jin   +7 more
wiley   +1 more source

Swelling‐Programmed Topographical Guidance for Dynamic Spheroid Self‐Assembly via a Mechanochemical Hydrogel Niche

Advanced Functional Materials, EarlyView.
A swelling‐programmed micropatterned hydrogel guides adherent cells through a controlled transition from cell–matrix anchoring to cadherin‐mediated cell–cell compaction, enabling rapid assembly of high‐viability spheroids with defined size and morphology.
Han Gyeol Nam, Seon Yeong Chae, Young Woo Kwon, Hye Jin Heo, Hye Rin Lee, Ji Hyeok Lee, Dong‐Wook Han, Yun Hak Kim, Suck Won Hong   +8 more
wiley   +1 more source

Intrinsic Photoactive Star ZnPc–Poly(glutamate) Nanoplatforms for Multimodal Glioblastoma Therapy and Brain‐Targeted Delivery

Advanced Functional Materials, EarlyView.
An intrinsic photoactive star‐shaped zinc phtalocyanine‐poly(L‐glutamic acid) (ZnPc‐PGA) nanoplatform for multimodal glioblastoma (GBM) therapy and brain‐targeted elivery. A ZnPc‐PGA‐based multifunctional theranostic nanocarrier platform enables image‐guided, multimodal GBM therapy. ZnPc‐PGA nanocarriers support the integration of fluorescence imaging,
Amina Benaicha‐Fernández, Teodora Ranđelović, Ana Armiñán, Víctor Sebastián, Manuel Fuentes, Cristián Huck‐Iriart, Esther Masiá, Paula Carrascosa‐Marco, Pablo Juanes‐Velasco, Snežana Đorđević, Clara Bayona, Ignacio Ochoa, Maria Medel, Inmaculada Conejos‐Sánchez, María J. Vicent   +14 more
wiley   +1 more source

Mechanoluminescent HOF Nanotransducers Enabled Sono‐Optogenetics in Parkinsonian Rats

Advanced Functional Materials, EarlyView.
We present a mechanoluminescent system utilizing porous hydrogen‐bonded organic frameworks (HOFs) as a toolkit for focused ultrasound‐triggered, non‐invasive light delivery to the deep brain in rats. This approach enables the specific activation of PV‐GPe neurons in dopamine‐depleted Parkinson's disease rat models, resulting in a comparable alleviation
Wenliang Wang, Ilya Pyatnitskiy, Yanshu Shi, Kai Wing Kevin Tang, Yi Xie, Sihan Yu, Thomas Wynn, Xiangping Liu, Ju‐Chun Hsieh, Jinmo Jeong, Weilong He, Brinkley Artman, Anakaren Romero Lozano, Xi Shi, Arjun Sangani, Lief E Fenno, Samantha Santacruz, Banglin Chen, Huiliang Wang   +18 more
wiley   +1 more source

Gene replacement therapy in a model of Charcot-Marie-Tooth 4C neuropathy. [PDF]

Brain, 2019
Schiza N, Georgiou E, Kagiava A, Médard JJ, Richter J, Tryfonos C, Sargiannidou I, Heslegrave AJ, Rossor AM, Zetterberg H, Reilly MM, Christodoulou C, Chrast R, Kleopa KA.   +13 more
europepmc   +1 more source