Optimization of AAV vectors to target persistent viral reservoirs
Gene delivery of antiviral therapeutics to anatomical sites where viruses accumulate and
persist is a promising approach for the next generation of antiviral therapies. Recombinant …
persist is a promising approach for the next generation of antiviral therapies. Recombinant …
Intrathecal sc-AAV9-CB-GFP: systemic distribution predominates following single-dose administration in cynomolgus macaques
EK Meseck, G Guibinga, S Wang… - Toxicologic …, 2022 - journals.sagepub.com
Biodistribution of self-complementary adeno-associated virus-9 (scAAV9)–chicken β-actin
promoter–green fluorescent protein (GFP) was assessed in juvenile cynomolgus macaques …
promoter–green fluorescent protein (GFP) was assessed in juvenile cynomolgus macaques …
HRD1-induced TMEM2 ubiquitination promotes ER stress-mediated apoptosis through a non-canonical pathway in intestinal ischemia/reperfusion
X Zhao, D Liu, Y Zhao, Z Wang, Y Wang, Z Chen… - Cell Death & …, 2024 - nature.com
Intestinal ischemia/reperfusion (I/R) injury is a typical pathological course in the clinic with a
high morbidity rate. Recent research has pointed out the critical role of ubiquitination during …
high morbidity rate. Recent research has pointed out the critical role of ubiquitination during …
[HTML][HTML] Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems
Gene therapy offers great promise in addressing neuropathologies associated with the
central and peripheral nervous systems (CNS and PNS). However, genetic access remains …
central and peripheral nervous systems (CNS and PNS). However, genetic access remains …
Life‐Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy
AK Johnson, VJ McCurdy… - Annals of …, 2023 - Wiley Online Library
Objective GM2 gangliosidosis is usually fatal by 5 years of age in its 2 major subtypes, Tay‐
Sachs and Sandhoff disease. First reported in 1881, GM2 gangliosidosis has no effective …
Sachs and Sandhoff disease. First reported in 1881, GM2 gangliosidosis has no effective …
Optogenetic control of the enteric nervous system and gastrointestinal transit
Introduction: There are limited effective therapies available for improving gastrointestinal (GI)
transit in mammals with intractable or chronic constipation. Current therapeutics to improve …
transit in mammals with intractable or chronic constipation. Current therapeutics to improve …
Adeno‐associated virus‐9 reverses delayed gastric emptying of solids in diabetic mice
X Li, S Ji, G Cipriani, ML Hillestad… - …, 2023 - Wiley Online Library
Background Gastroparesis is defined by delayed gastric emptying (GE) without obstruction.
Studies suggest targeting heme oxygenase‐1 (HO1) may ameliorate diabetic gastroparesis …
Studies suggest targeting heme oxygenase‐1 (HO1) may ameliorate diabetic gastroparesis …
Fecal supernatants from dogs with idiopathic epilepsy activate enteric neurons
K Elfers, A Watanangura, P Hoffmann… - Frontiers in …, 2024 - frontiersin.org
Introduction Alterations in the composition and function of the gut microbiome have been
reported in idiopathic epilepsy (IE), however, interactions of gut microbes with the enteric …
reported in idiopathic epilepsy (IE), however, interactions of gut microbes with the enteric …
Adeno-associated virus vector intraperitoneal injection induces colonic mucosa and submucosa transduction and alters the diversity and composition of the faecal …
LT Ma, JX Lian, Y Bai, MJ Shang, ZZ Zhang… - Frontiers in Cellular …, 2022 - frontiersin.org
Background Viral vector technology, especially recombinant adeno-associated virus vector
(rAAV) technology, has shown great promise in preclinical research for clinical applications …
(rAAV) technology, has shown great promise in preclinical research for clinical applications …
Transduction of systemically administered adeno-associated virus in the colonic enteric nervous system and c-kit cells of adult mice
L Wang, PQ Yuan, C Challis… - Frontiers in …, 2022 - frontiersin.org
Systemic delivery of adeno-associated virus (AAV) vectors transduces the enteric nervous
system. However, less is known on the mapping and morphological and neurochemical …
system. However, less is known on the mapping and morphological and neurochemical …