Results 181 to 190 of about 110,488 (256)

AAVrh10 gene therapy ameliorates central and peripheral nervous system disease in canine globoid cell leukodystrophy (Krabbe disease) [PDF]

, 2018
et al,, Jiang, Xuntain, Ory, Daniel S
core   +1 more source

Image Analysis Platform for Comprehensive Quantification of Extracellular Vesicle Morphology

PROTEOMICS, EarlyView.
ABSTRACT Extracellular vesicles (EVs) are membrane‐encapsulated nanoparticles with key roles in cell biology and growing importance in biomedicine. However, EVs are challenging to characterize because they are highly heterogeneous. Single EV analysis methods are valuable tools for addressing this challenge.
Alexander Spark, Nan Jiang, Benjamin Purnell, Mihajlo Radmilovic, Andras Saftics, Eugenia Romano, Tijana Jovanovic‐Talisman, Alexandre Kitching   +7 more
wiley   +1 more source

Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy. [PDF]

Nat Med
Buddle S, Brown LK, Morfopoulou S, Torres Montaguth OE, Scoto M, Herder V, Dhawan A, Brown JR, Atkinson L, Kopec A, Davis D, Storey N, Campos L, Sebire N, Macpherson H, Lee J, Orton R, Baranello G, Asamaphan P, Ilia G, Karda R, Belfield H, ISARIC4C Investigators, Counsell J, Waddington SN, Thomson EC, Muntoni F, Breuer J.   +27 more
europepmc   +1 more source

Noise‐Induced Hearing Loss: From Pathological Mechanisms to Therapeutic Interventions

Sensory Neuroscience, EarlyView.
ABSTRACT Noise‐induced hearing loss (NIHL), a significant non‐genetic form of hearing impairment, is primarily managed through the use of hearing aids and cochlear implants. However, the fundamental pathological mechanisms underlying NIHL remain inadequately addressed.
Shiqi Huang, Yu Xiao, Jiaxin Liu, Wen Jiang, Weiwei Shi, Hanbing Zhang, Xiaolong Fu, Yunhao Wu   +7 more
wiley   +1 more source

Adeno-Associated Virus-Based Gene Therapy for Lafora Disease in <i>Epm2b</i>-Deficient Mice. [PDF]

Int J Mol Sci
Zafra-Puerta L, Iglesias-Cabeza N, Sanz P, García-Gimeno MA, Sánchez-Martín G, Sánchez MP, Serratosa JM.   +6 more
europepmc   +1 more source

Rapid generation of prion disease models using AAV‐delivered PrP variants in knockout mice

Brain Pathology, EarlyView.
We developed a rapid AAV‐based system to generate prion disease models in weeks rather than months. Following systemic AAV9P31 delivery of modified PrP to knockout mice, we achieved brain‐wide expression and successful propagation of both classical (RML) and atypical (GSS‐A117V) prion strains.
Maitena San‐Juan‐Ansoleaga, Eva Fernández‐Muñoz, Jorge M. Charco, Enric Vidal, Diego Herrero‐Martínez, Josu Galarza‐Ahumada, Cristina Sampedro‐Torres‐Quevedo, Samanta Giler, Mariví Geijo, Gloria González‐Aseguinolaza, Hasier Eraña, Joaquín Castilla   +11 more
wiley   +1 more source

Insights into Adeno-Associated Virus Capsid Charge Heterogeneity. [PDF]

Anal Chem
Wu J, Draper BE, Jarrold MF, Heger CD.
europepmc   +1 more source

The DNA/RNA autophagy protein SIDT2 as a novel neuropathological hallmark in Huntington disease

Brain Pathology, EarlyView.
SIDT2‐immunoreactive inclusions are observed in the striatum, cerebral cortex, and hypothalamus in HD cases with different Vonsattel grades, and the frequency of SIDT2‐immunoreactive inclusions is associated with longer CAG repeats in the huntingtin gene.
Sanaz Gabery, Sofia Bergh, Chrisovalantou Huridou, Rachel Y. Cheong, Barbara Baldo, Paul Günther Scheunemann, Marie‐Louisa Schoebel, Linda Holmquist Mengelbier, Elisabet Englund, Catriona McLean, Carsten Saft, Deniz Kirik, Maria Björkqvist, Glenda Halliday, Elisabeth Petrasch‐Parwez, Huu Phuc Nguyen, Jonasz Jeremiasz Weber, Åsa Petersén   +17 more
wiley   +1 more source

Suppressing microtubule detyrosination augments adeno-associated virus 2 endosomal escape and gene delivery. [PDF]

J Cell Sci
Tripathi S, Huda S, Kar J, Chandra D, Jayandharan GR, Mohan N.   +5 more
europepmc   +1 more source

PDE4D and PDE3B orchestrate distinct cAMP microdomains in 3T3‐L1 adipocytes

British Journal of Pharmacology, EarlyView.
Basal conditions: •Ins/PDE3B lowers cytoplasmic cAMP (cyt‐cAMP) without affecting plasma membrane cAMP (pm‐cAMP). •Insulin decreases lipid droplet cAMP (LD‐cAMP) independent of PDE3B. •FGF1/PDE4D modestly reduces both cyt‐ and pm‐cAMP, while PDE4D alone can modulate LD‐cAMP. ISO stimulation: •Ins/PDE3B has minimal impact on cyt‐cAMP.
Johannes Krier, David Spähn, David Arturo Juarez Lopez, Judith L. Nono, Judith Seigner, Lisa Jacob, Siegfried Ussar, Robert Lukowski, Andreas L. Birkenfeld, Gencer Sancar   +9 more
wiley   +1 more source