Engineered virus-like particles for effificient in vivo delivery of therapeutic proteins 半胱氨酸碱基编辑器(CBEs)和腺嘌呤碱基编辑器(ABEs)可以共同应用于更正绝大多数已知的由单核苷酸突变导致的疾病,并在小鼠和非人类灵长类动物中进行了测试,证实体内碱基编辑具有成为一种治疗策略的潜力。 体内碱基编辑技术的广泛...
In vitro and in vivo delivery of therapeutic proteins using cell penetrating peptides. Peptides 87, 50-63 (2016).Bolhassani, A.; Jafarzade, B.S.; Mardani, G. In vitro and in vivo delivery of therapeutic proteins using cell penetrating peptides. Peptides 2017, 87, 50-63....
Protein delivery with cell-penetrating peptide is opening up the possibility of using targets inside cells for therapeutic or biological applications; however, cell-penetrating peptide-mediated protein delivery commonly suffers from ineffective endosomal
Therapeutic application of virus-based delivery systems often implies a change of the tropism of these vectors. This can be achieved by insertion of polype... S Gleiter,H Lilie - 《Protein Science》 被引量: 48发表: 2001年 IN VIVO ACTIVATION OF ANTIGEN PRESENTING CELLS FOR ENHANCEMENT OF IMMU...
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins Cell, 185 (2022), pp. 250-265.e16, 10.1016/j.cell.2021.12.021 View PDFView articleView in ScopusGoogle Scholar Bareford and Swaan, 2007 L.M. Bareford, P.W. Swaan Endocytic mechanisms for targeted drug...
Amaurosis 10, as well as CRISPR/Cas9-edited T cells designed to target and destroy cancer cells. Further advances in therapeutic genome editing will rely on a safe and more efficient method ofin vivoCRISPR/Cas9 delivery and improved efficiency of homology-directed repair for site-specific gene ...
D. et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl Acad. Sci. USA 93, 14082–14087 (1996). ADS CAS PubMed PubMed Central Google Scholar Nathwani, A. C. et al. Long-term safety and efficacy of factor ...
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Over the past few years, clinical translation of bench research resulted in the first examples of therapeutic success for the field of gene transfer. Adeno-associated virus (AAV) vectors are among the most suitable tools for in vivo gene delivery given their superior efficiency of transduction of...
Although several studies have since attempted to use exosomes to deliver therapeutic siRNAs and miRNAs,14,15 they all have to harvest huge amounts of exosomes from cell culture before in vivo delivery, making large scale clinical translation unaffordable.16 Moreover, the fragility and complicated ...