Viral vectors are an integral delivery vehicle in modern medicine and are used in everything from oncolytic viruses to vaccines and gene therapies. With two gene therapies approved by regulatory agencies in 2021 and more in 2022, these therapies are delivering on their promise despite concerns on...
Vectors based on Adeno-associated virus (AAV) and Lentivirus (LV) stand as ideal tools for gene delivery, however they do not work in cardiac ECs.To enhance the permissiveness of EC, we optimized and performed high-throughput screeningonadult cardiac ECsusing a library...
Working at VIVEbiotech, which is fully specialized on lentiviral vectors, I have observed this firsthand in very advanced fields, such as cell and gene therapy. In this industry, the effort is focused on improving the bioprocess in such a way that the manufacturing ...
Viral vectors (e.g. AAV) are the most common type of delivery; however, they possess some limitations such as small insertion size, high carcinogenesis risk, and immune system stimulation [168, 169]. Nanotechnology can contribute with new delivery alternatives for the exploitation of CRISPR-based...
68 we now wish to develop optimized tyrosinemutant AAV3–melittin vectors under the control of a 根据我们的早先研究以包含melittin基因的再组合腺病毒传染媒介驾驶由肝癌cellspecific促进者达到肝癌细胞具体杀害体外和体内,我们现在希望开发优化tyrosinemutant AAV3-melittin传染媒介受肝癌细胞具体促进者的控制有选择...
In parallel, due to the limited packaging capacity of viral vectors, transport and expression of shortened forms of dystrophin, the so-called mini- or microdystrophins, have been developed for gene therapy (Belanto et al., 2014, Barthélémy and Wein, 2018). However, the precise consequences ...
In the 37 years since Samulski cloned the first AAV, which came to be known as AAV2, a number of other types have emerged. There's estimated to be around a dozen that can infect human cells, and a number of these are being tested as gene therapy vectors for different diseases. The ...
medicine and developing gene therapies, we have made tremendous progress -from the early days of exploring viruses as possible delivery methods to where we are today, with multiple FDA-approved gene therapies that harness the power of adeno-associated virus (AAV) vectors for therapeutic potential. ...