The fact that it is a monogenic disease and that PiZ, the most frequent allele, is a well-characterized point mutation produced by a G > A substitution at codon 342 (Glu342Lys) in exon 5 of SERPINA1 gene make AATD a suitable target for gene therapy [114]. Overall, two different approa...
Codon Optimization Tool Company Start Order Start Order Synthetic Controls Genes Oligo Pools Custom Vectors Antibodies Get Quote Libraries NGS Genes Oligo Pools NGS Variant Libraries Synthetic Controls Antibody Discovery IVDR Solutions Genes...
It is a T2 segregated line validated as T-DNA-free by conventional PCR covering the entire binary plasmids used for Agrobacterium-mediated transformation. The edited allele contains a single-base insertion resulting in a premature stop codon just before the CaMBD domain, eliminating it from the ...
but can also inhibit survivin cell cycle-regulation, mRNA stability and translation efficiency. This is because the -43 to +1 regulatory sequence has cell cycle regulatory DNA elements and is transcribed into survivin mRNA as
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The KRAS protein is a GTPase that transmits intracellular signals upon binding to GTP [2], [3]. Under physiologic conditions, this system is maintained by guanine nucleotide exchange factors, favoring the GDP-bound inactive form. However, KRAS mutations promote maintenance of the GTP-bound active...
The strong safety profile of MVA-vectored vaccines as well as its history of safe use as smallpox vaccine have stagnated research into aspects of the biology of the vector that are still poorly understood but also relevant for further optimization of the safety of MVA. Thus, there is an ...