Neurotrophins as Potential Gene Therapy Targets for Huntington's Disease [0.03%]
神经生长因子作为亨廷顿舞蹈病的基因治疗靶点的潜力
Sagor Kumar Roy,Ashima Barman,Kumary Labone Sarkar et al.
Sagor Kumar Roy et al.
"Huntington's disease" (HD) is an autosomal dominant hereditary neurodegenerative disease characterized by defects in efferent striatal neurons, cortical neurons, and the basal ganglia. The pathogenesis of HD is still unclear, and there is ...
Targeting Undruggable Proteins: The siRNA Revolution Beyond Small Molecules - Advances, Challenges, and Future Prospects in Therapeutic Innovation [0.03%]
靶向不可成药蛋白质:小分子之后的siRNA革命——治疗创新领域的进展、挑战与未来前景
Sk Ashif Jan,Abhijit Debnath,Rajesh Kumar Singh et al.
Sk Ashif Jan et al.
The field of drug discovery has long been challenged by the existence of "undruggable" proteins - targets that have resisted traditional small molecule approaches due to their structural or functional characteristics. This review explores t...
Synergizing CRISPR-Cas9 with Advanced Artificial Intelligence and Machine Learning for Precision Drug Delivery: Technological Nexus and Regulatory Insights [0.03%]
CRISPR-Cas9与先进人工智能和机器学习在精准药物递送中的协同作用:技术交汇点与监管见解
Amrita Arup Roy,Rahul Pokale,Anoushka Mukharya et al.
Amrita Arup Roy et al.
The evolution of genetic exploration tools, from laborious methods like radiationinduced mutations to the transformative CRISPR-Cas9 system, has fundamentally reshaped genetic research and gene editing capabilities. This journey, initiated ...
Molecular Insights into RNA Modifications and their Role in Shaping Immune Responses and Tumor Microenvironments [0.03%]
RNA修饰的分子见解及其在塑造免疫反应和肿瘤微环境中的作用
Ashiq Ali,Tehreem Ajmal,Aisha Khatoon et al.
Ashiq Ali et al.
RNA modifications play crucial roles in immune system development and function, with dynamic changes essential for diverse cellular processes. Innovative profiling technologies are invaluable for understanding the significance of these modi...
Exploring Genetic and Receptor-Based Dopaminergic Strategies for Antidepressant Drug Development [0.03%]
基于遗传和受体的多巴胺能策略的抗抑郁药物开发研究
Arzoo Pannu,Ramesh K Goyal
Arzoo Pannu
The dopamine (DA) system is central to mood regulation, motivation, and reward processing, making it a critical focus for understanding Major Depressive Disorder (MDD). While the dopaminergic system's role in MDD pathophysiology has been ac...
Plasmalogens Activate AKT/mTOR Signaling to Attenuate Reactive Oxygen Species Production in Spinal Cord Injury [0.03%]
血浆脂醇活化AKT/mTOR信号减轻脊髓损伤后的活性氧生成
Mengdan Cheng,Yan Gao,Yiqing Wu et al.
Mengdan Cheng et al.
Background: Plasmalogens, the primary phospholipids in the brain, possess intrinsic antioxidant properties and are crucial components of the myelin sheath surrounding neuronal axons. While their neuroprotective effects ha...
siRNA Knocking Down in HepG2 Cells Identifies PFKFB4 and HNF4α as Key Genes Important for Cancer Cell Survival [0.03%]
siRNA敲除在HepG2细胞中鉴定PFKFB4和HNF4α为对癌细胞生存重要的关键基因
Amer Imraish,Walhan Alshaer,Tuqa Abu-Thiab et al.
Amer Imraish et al.
Introduction: Liposomes are versatile delivery systems for encapsulating small interfering RNAs (siRNAs) because they enhance cellular uptake and gene silencing. This study compares the new liposome formula to commercial ...
Development of Lentiviral Packaging Cells and Scale Up of Production to Meet the Growing Demand in Cell and Gene Therapy [0.03%]
lentivirus包装细胞的开发及其在细胞和基因治疗需求不断增长情况下的生产工艺放大
Yasemin van Heuvel,Jörn Stitz
Yasemin van Heuvel
Gamma-Retroviral (RVVs) and lentiviral vectors (LVVs) represent indispensable tools in somatic gene therapy, mediating the efficient, stable transfer of therapeutic genes into a variety of human target cells. LVVs, in contrast to RVVs, are ...
O-substituted Tertiary Amine-chitosans as Promising Nanocarriers for siRNA Delivery [0.03%]
O-取代的叔胺壳聚糖作为siRNA递送的有前景的纳米载体
André Miguel Martinez Júnior,Vera Aparecida de Oliveira,Marcio José Tiera
André Miguel Martinez Júnior
Introduction: The clinical translation of chitosan-based formulations for siRNA delivery has been partially limited by their poor stability/solubility at physiological conditions, although they have good biocompatibility ...
CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges [0.03%]
CRISPR/Cas系统:改善单基因人类疾病的治疗递送的有效策略;当前应用与挑战
Fatemeh Saberi,Zeinab Yousefi-Najafabadi,Forough Shams et al.
Fatemeh Saberi et al.
The 5,000 to 8,000 monogenic diseases are inherited disorders leading to mutations in a single gene. These diseases usually appear in childhood and sometimes lead to morbidity or premature death. Although treatments for such diseases exist,...