Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy [0.03%]
系统给药mRNA-LNP改善脊髓性肌萎缩症小鼠模型的存活率
Marco D Carpenter,Nicole Kus,Rosa B Choi et al.
Marco D Carpenter et al.
Base editing provides a unique opportunity for the permanent correction of central nervous system (CNS) diseases with early onset pathology. While lipid nanoparticles (LNPs) have emerged as an effective messenger RNA (mRNA) delivery system ...
A potency and toxicology study on tolerability of cryopreserved mesenchymal stem cell product with DMSO in septic mice and nude rats [0.03%]
评估含DMF的冻存间充质干细胞产品在败血症小鼠和裸鼠体内耐受性的药效学和毒理学研究
Yan Wang,Glinton Hanover,Aidan B P Murray et al.
Yan Wang et al.
Mesenchymal stem/stromal cell (MSC)-based therapies have emerged as a promising treatment for sepsis, supported by encouraging preclinical data. DMSO has been a widely used cryoprotectant in cell-based therapeutic products; however, whether...
Therapeutic mRNA delivery of CRISPR-Cas9 to the trabecular meshwork reverses ocular hypertension in myocilin glaucoma [0.03%]
通过小梁网递送治疗性mRNA传递CRISPR-Cas9逆转肌苷诱导的青光眼所致的眼压增高
Sam Yacoub,Balasankara Reddy Kaipa,Linya Li et al.
Sam Yacoub et al.
Mutations in the myocilin gene (MYOC) are the leading genetic cause of primary open angle glaucoma (POAG), the most common glaucoma type. These mutations trigger a toxic gain-of-function phenotype, causing the misfolded MYOC protein to accu...
Carrie L Cummings,Sirkka B Stephan,Katelyn Fitzgerald et al.
Carrie L Cummings et al.
Hematopoietic stem cell (HSC) gene therapies hold immense potential for treating a growing list of genetic disorders, but the field has reached an inflection point where novel technologies are needed to overcome significant challenges, incl...
Heavy-chain immunoglobulin locus editing in rhesus macaque B cells to confer antibody production [0.03%]
利用基因编辑技术使食蟹猴B细胞重链抗体表达单元活化以产生全人源单克隆抗体技术研发项目
Anne-Sophie Kuhlmann,Nawal Madkhali,Eliana Moskovitz et al.
Anne-Sophie Kuhlmann et al.
Antibody-based immunotherapies are promising; however, their application remains limited to acute diseases due to rapid clearance of antibodies in vivo. Some chronic conditions could benefit from sustained therapeutic antibody expression. O...
Karoliina Tuomela,Emily S Y Leong,Manjurul Haque et al.
Karoliina Tuomela et al.
Adoptive cell therapy using regulatory T cells (Tregs) is a promising approach to suppress immune responses in autoimmunity and transplantation, but it is challenging to expand pure and optimally suppressive cells. Lactic acid (LA) is assoc...
Efficient kidney gene transfer and proximal tubule transduction using self-complementary AAV.cc47 vectors [0.03%]
自互补AAV.cc47载体的高效肾脏基因转移及近端小管转导
Jennifer L Peek,Alan Rosales,Julie Qi et al.
Jennifer L Peek et al.
Gene delivery to critical cell types within the kidney can enable preclinical evaluation of gene therapies for kidney disease. The novel adeno-associated virus AAV.cc47 was discovered after sequential evolution in mice, pigs, and macaques a...
Chronologically distributed transfection improves AAV2 and AAV2/8 capsid filling and reveals assembly schedule divergence [0.03%]
时间分布转染可提高AAV2和AAV2 / 8衣壳填充并揭示装配时间表的分歧
Qiantong Chen,Chae Hyon Lee,Robert Whitfield et al.
Qiantong Chen et al.
Adeno-associated virus (AAV) gene therapy vectors often suffer from low capsid filling, resulting in high proportions of empty capsids that reduce efficacy and complicate manufacturing processes. This study investigates whether chronologica...
Generation of high-quality single-stranded DNA for full-length and truncated genome standards of recombinant adeno-associated viruses [0.03%]
用于重组腺相关病毒全长和截短型基因组标准的高质单链DNA的制备
Julia Manz,Raphael Ruppert,Markus Haindl et al.
Julia Manz et al.
The integrity of the single-stranded DNA (ssDNA) genome of recombinant adeno-associated viral vectors (rAAVs) for gene therapy applications is a critical quality attribute. To monitor this attribute, robust analytical methods, such as long-...
Landscape of ex vivo gene therapies: Technological trends and future prospects [0.03%]
体外基因治疗全景：技术趋势和未来前景
Aline Almeida-Oliveira,Martín Hernán Bonamino,Marisol Simões et al.
Aline Almeida-Oliveira et al.
Ex vivo gene therapy, a revolutionary approach involving extraction, genetic modification, and infusion of cells, is rapidly evolving, making it difficult to track its advancements. Strategic foresight methodologies play a crucial role in u...