Addressing the Challenges of Treating Patients with Heterozygous Gain of Function Mutations [0.03%]
杂合子功能获得性突变患者治疗面临的挑战
Stanley T Crooke
Stanley T Crooke
Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells [0.03%]
反义寡核苷酸STK-002可增加OPA1在视网膜中的含量并改善线粒体功能障碍型常染色体显性遗传 optic atrophy 细胞
Aditya Venkatesh,Taylor McKenty,Syed Ali et al.
Aditya Venkatesh et al.
Autosomal dominant optic atrophy (ADOA) is an inherited optic neuropathy most frequently associated with OPA1 mutations. Most variants result in haploinsufficiency, and patient cells express roughly half of the normal levels of OPA1 protein...
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy [0.03%]
近似序列同源性不能保证siRNA在物种间的有效性
Iris Valeria Rivera Flores,Kathryn Monopoli,Samuel Jackson et al.
Iris Valeria Rivera Flores et al.
Small interfering RNAs (siRNAs) represent a novel class of drugs capable of potent and sustained modulation of genes across various tissues. Preclinical development of siRNAs necessitates assessing efficacy and toxicity in animal models. Wh...
Mass Spectrometry as a Quantitative Tool for SpCas9 sgRNA Quality Control [0.03%]
基于SpCas9的sgRNA质控的定量分析方法mass spectrometry
Juan Daniel Avila,Puzhou Wang
Juan Daniel Avila
Mass spectrometry (MS) has long been used for quality control of oligonucleotide therapeutics, including single-guide RNAs (sgRNAs) for clustered regularly interspaced short palindromic repeats techniques. However, the application of MS is ...
A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides [0.03%]
寡核苷酸药物临床前开发中的化学考量指南
Daniel O&#x;Reilly,Willeke van Roon-Mom,Annemieke Aartsma-Rus;N = Collaborative
Daniel O&#x;Reilly
Oligonucleotide therapeutics, a pioneering category of modern medicinal drugs, are at the forefront of utilizing innate mechanisms to modulate gene expression. With 18 oligonucleotide-based FDA-approved medicines currently available for tre...
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon [0.03%]
Tricyclo-DNA修饰反义寡核苷酸增加与DMD靶标外显子亲和力不会造成外显子跳过水平高估
Mathilde Doisy,Ophélie Vacca,Amel Saoudi et al.
Mathilde Doisy et al.
Antisense oligonucleotides (ASO) are very promising drugs for numerous diseases including neuromuscular disorders such as Duchenne muscular dystrophy (DMD). Several ASO drugs have already been approved by the US Food and Drug Administration...
Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity [0.03%]
用于调节CRISPR-Cas9特异性的肽核酸的设计与合成
Kelly E W Carufe,Nicholas G Economos,Peter M Glazer
Kelly E W Carufe
Although CRISPR-Cas9 gene therapies have proven to be a powerful tool across many applications, improvements are necessary to increase the specificity of this technology. Cas9 cutting in off-target sites remains an issue that limits CRISPR'...
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo [0.03%]
体内实验中由信使rna核聚集引起的短干扰rnas沉默突变体亨廷顿病信使rna和蛋白质的差异
Sarah Allen,Daniel O&#x;Reilly,Rachael Miller et al.
Sarah Allen et al.
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG repeat expansion in the first exon of the huntingtin gene (HTT). Oligonucleotide therapeutics, such as short interfering RNA (siRNA), reduce levels o...
Characterization of the TLR9-Activating Potential of LNA-Modified Antisense Oligonucleotides [0.03%]
具有TLR9激活作用的LNA修饰反义寡核苷酸的特点分析研究
Irene Riera-Tur,Julia Hinterdobler,André Maaske et al.
Irene Riera-Tur et al.
Early characterization of the immunostimulatory potential of therapeutic antisense oligonucleotides (ASOs) is crucial. At present, little is known about the toll-like receptor 9 (TLR9)-mediated immunostimulatory potential of third-generatio...
Preclinical Pharmacokinetics in Tumors and Normal Tissues of the Antigene PNA Oligonucleotide MYCN-Inhibitor BGA002 [0.03%]
抗基因PNA寡核苷酸MYCN抑制剂BGA002的肿瘤与正常组织药代动力学研究
Anna Lisa Scardovi,Damiano Bartolucci,Luca Montemurro et al.
Anna Lisa Scardovi et al.
Although MYCN has been considered an undruggable target, MYCN alterations confer poor prognosis in many pediatric and adult cancers. The novel MYCN-specific inhibitor BGA002 is an antigene peptide nucleic acid oligonucleotide covalently bound...