Effective Treatment of Colq-Deficient Mice with Adeno-Associated Virus Type Rh74-Mediated Gene Therapy [0.03%]
利用Rh74腺相关病毒介导的基因治疗有效治疗Colq缺陷型小鼠
Abigail McInnes,Jaime N Young,Anahid S Aivazian et al.
Abigail McInnes et al.
Mutations in human COLQ, which encodes the collagen-like tail subunit (ColQ) of asymmetrical acetylcholinesterase (AChE), cause congenital myasthenic syndrome (CMS) with deficiency of end plate AChE. A valuable animal model of COLQ-CMS is t...
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye [0.03%]
SNAC：非人灵长类动物眼睛衣壳分布的单核基因组图谱
Rachna Manek,Eugenia Lyashenko,Andre H Kurlovs et al.
Rachna Manek et al.
Adeno-associated virus (AAV) vectors have emerged as the leading gene therapy vehicle due to their favorable safety profile and sustained payload expression. Approved therapies such as voretigene neparvovec (Luxturna) and omnasemnogene abep...
Pre-Existing Anti-Adeno-Associated Virus Immunity in Gene Therapy: Mechanisms, Challenges, and Potential Solutions [0.03%]
基因治疗中腺相关病毒预先存在的免疫反应：机制、挑战及潜在解决方案
Godwin I Iroanya,Pradeep N Subramanyam,Kevin D Wells et al.
Godwin I Iroanya et al.
Adeno-associated virus (AAV) vectors have emerged as versatile and promising tools in gene therapy due to their favorable safety profile, broad tissue tropism, and long-term gene expression. However, pre-existing immunity, especially in the...
Nicolas Lonfat,Laura Moreno-Leon,Claudio Punzo et al.
Nicolas Lonfat et al.
Inherited and complex retinal degenerative diseases, such as retinitis pigmentosa, age-related macular degeneration, and glaucoma, represent a significant global burden of irreversible vision loss. Due to immense genetic and clinical hetero...
Vertex, Enlaza Launch Up-to-$2B Collaboration to Improve CASGEVY® Conditioning [0.03%]
Vertex和Enlaza启动高达20亿美元的合作以改进CASGEVY®预处理方案
Alex Philippidis
Alex Philippidis
Lentiviral Gene Delivery Rescues Ciliary Defects in Patient-Derived Airway Organoids from Primary Ciliary Dyskinesia [0.03%]
慢病毒基因传递可修复原发性纤毛运动障碍患者衍生呼吸道类器官中的纤毛缺陷
Chunxiao Huo,Ting Luo,Lei Wu et al.
Chunxiao Huo et al.
Primary ciliary dyskinesia (PCD) is a genetic disorder characterized by defective ciliary motility, leading to recurrent respiratory infections and chronic airway damage. Gene therapy holds promise for treating PCD, but its effectiveness in...
Preclinical Assessment of Antibody Responses to Adeno-Associated Virus (AAV) Vector-Based Capsids of AAV2, AAV5, AAV8, or AAV9 in Laboratory Cynomolgus Macaques (Macaca fascicularis) of Asian or Mauritian Origin [0.03%]
实验室恒河猴的小型非人灵长类动物AAV衣壳的腺相关病毒(AAV)载体基于AAV2，AAV5，AAV8或AAV9的抗体反应的临床前评估
Betina Pajaziti,Ulf Michgehl,Dragica Blazevic et al.
Betina Pajaziti et al.
Adeno-associated virus (AAV)-based vectors are the most commonly used vectors for gene therapy. Wild-type AAV infections occur widely in humans and nonhuman primates (NHPs), and an accurate assessment of preexisting AAV antibodies is crucia...
Targeting Amyotrophic Lateral Sclerosis with Gene Therapy: From Silencing Genes to Enhancing Neuroprotection [0.03%]
针对肌萎缩侧索硬化症的基因治疗：从基因沉默到增强神经保护作用
Sergi Verdés,Xavier Navarro,Assumpció Bosch
Sergi Verdés
Gene therapy is emerging as a transformative approach for treating amyotrophic lateral sclerosis (ALS), a progressive and fatal neurodegenerative disease. While gene replacement has shown a groundbreaking success in spinal muscular atrophy,...
Jimena Pérez-Maroto,Laura Sepp-Lorenzino,Diego Castaño-Esteban et al.
Jimena Pérez-Maroto et al.
Rare diseases are serious and often chronic conditions that affect a small number of individuals. However, with over 7,000 rare diseases identified, their cumulative global numbers and impact are substantial. A considerable proportion of th...