Results 231 to 240 of about 172,174 (293)

Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial. [PDF]

EBioMedicine
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S.   +38 more
europepmc   +1 more source

Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy. [PDF]

J Inherit Metab Dis, 2018
Brooks ED, Landau DJ, Everitt JI, Brown TT, Grady KM, Waskowicz L, Bass CR, D'Angelo J, Asfaw YG, Williams K, Kishnani PS, Koeberl DD.   +11 more
europepmc   +1 more source

Chamber‐Specific Decellularized Extracellular Matrices Differentially Modulate Cardiomyocyte Subtypes to Drive Engineered Heart Tissue Development and Function

Advanced Healthcare Materials, EarlyView.
Chamber‐specific decellularized extracellular matrices (ECMs) were developed, preserving native proteomic profiles of ventricular and atrial myocardium. These innate biochemical cues differentially modulate cardiomyocyte subtypes to drive engineered heart tissue development and function, highlighting the importance of incorporating regional ECM cues in
Dong Gyu Hwang, Myungji Kim, Hwanyong Choi, Jinseon Park, Minji Kim, Donghwan Kim, Bo Ram Lee, Jinah Jang   +7 more
wiley   +1 more source

A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1. [PDF]

Mol Ther Methods Clin Dev, 2018
Shababi M, Villalón E, Kaifer KA, DeMarco V, Lorson CL.   +4 more
europepmc   +1 more source

Coaxially Electrospun Myocardial dECM‐ Based Nanofibrous Scaffolds Demonstrate Enhanced Cardiomyocyte Biocompatibility

Advanced Healthcare Materials, EarlyView.
Our coaxial electrospinning system is capable of incorporating decellularized myocardial extracellular matrix in a core‐sheath configuration, creating nanofibrous scaffolds. These scaffolds, in turn, support the maintenance of functional cardiomyocytes adhered to the surface of these bioactive scaffolds.
Dhanusha N. Rajapakse, Mahtab Khodadadi, Kiran M. Ali, Suh Hee Cook, Bryson T. Proctor, Tanya Upadhyay, Daxian Zha, Fatema Tuj Jahura, Marziyeh Boosaliki, Jessica M. Gluck   +9 more
wiley   +1 more source

Flash Assembloids: A Rapid Biofabrication of a Platform for Modeling Early Glioblastoma Invasion at the Glioblastoma–Brain Organoid Interfaces

Advanced Healthcare Materials, EarlyView.
This study presents a bioengineered assembloid (ASM) system combining glioblastoma (GBM) cells in oxidized alginate (OA) microgels with dorsal organoids (DOs). This model simulates brain tumor‐host interactions, revealing enhanced GBM invasion, altered gene expression, and aggressive infiltration patterns, demonstrating ASM as a valuable platform for ...
Chao Liang, Henry Robert Howard, Shihui Chen, Won‐Young Choi, Summer Cao, Arthur Chien, Kevin Zou, Michael Kassiou, Fabien Delerue, Ho Sang Jung, Yeonju Park, Young Mee Jung, Yong‐Dae Kwon, Karrie M. Kiang, Gilberto Ka‐Kit Leung, Ryohichi Sugimura, Ann‐Na Cho, Sang Jin Lee   +17 more
wiley   +1 more source

Multidimensional Cellular Micro‐Compartments to Model Invasive Lobular Carcinoma Dormancy

Advanced Healthcare Materials, EarlyView.
Invasive lobular carcinoma (ILC) is an understudied subtype of breast cancer that is susceptible to late recurrences. In this study, micro‐compartmentalization techniques spanning multiple dimensions, including 2D, pseudo‐3D, and 3D, are integrated to uncover the mechanisms underlying ILC dormancy, revealing the central role of p27Kip1.
Xilal Y. Rima, Sarmila Majumder, Divya S. Patel, Chunyu Hu, Hong Li, Xin Huang, Kim Truc Nguyen, Jacob Doon‐Ralls, Chiranth K. Nagaraj, Mangesh D. Hade, Setty M. Magaña, Eswar Shankar, Xiaoli Zhang, Daniel G. Stover, Bhuvaneswari Ramaswamy, Eduardo Reátegui   +15 more
wiley   +1 more source

Photothermal‐Activated Antibacterial Amyloid‐Polyphenol‐Iron Hydrogels for Synergistic Wound Healing

Advanced Healthcare Materials, EarlyView.
We report a thermally triggered supramolecular hydrogel (LTFe) formed by Fe3+ and tannic acid on lysozyme amyloid fibrils. Fe3+ enables rapid gelation and photothermal transduction with an efficiency of 88.56%. The LTFe hydrogel exhibits excellent biocompatibility, potent antibacterial activity against E. coli and S.
Di Wu, Jiangtao Zhou, Yang Shen, Qiyao Sun, Tong Li, Xiaoyang Zou, Bin Liu, Hongshan Liang, Raffaele Mezzenga   +8 more
wiley   +1 more source

Functional Blood‐Brain Barrier Crossing by Biomimetic M13 Phage Vectors for Targeted Neuronal Delivery

Advanced Healthcare Materials, EarlyView.
This study investigates the M13 bacteriophage as a biomimetic nanovector capable of crossing in vitro models of the blood–brain barrier. By exploiting peculiar transcellular pathways, M13 avoids lysosomal degradation and preserves its structural integrity and functionality.
Silvia Vercellino, Lucia Pappagallo, Francesca De Chirico, Luca Boselli, Hazem Ahmed, Luca Spagnoletti, Giovanni Pittaluga, Paolo Emidio Costantini, Matteo Calvaresi, Pier Paolo Pompa, Alberto Danielli, Fabio Benfenati, Valentina Castagnola   +12 more
wiley   +1 more source

Hydrogel Microtube Drug Carrier for Catheter‐Based Intravascular Therapy

Advanced Healthcare Materials, EarlyView.
This paper proposes hydrogel microtube carriers composed of barium alginate gel, enabling long‐term vascular retention without interrupting blood flow. Intravascular treatments have long been attracting attention for therapeutic efficacy, yet practical delivery methods remained unestablished.
Shota Sato, Hiroki Ohta, Naoki Takakura, Kentaro Kasa, Teppei Komatsu, Dai Kejia, Misato Yoshioka, Hirotaka James Okano, Hiroaki Onoe   +8 more
wiley   +1 more source