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Molecular Therapy is a scientific journal, published by Cell Press, that aims to develop and explore "molecular and cellular therapeutics to correct genetic and acquired diseases". [ 1 ] The founder of the journal and its Editor-in-Chief in the first five years was Inder Verma .
The DNA binding domain contains a repeated highly conserved 33–34 amino acid sequence with divergent 12th and 13th amino acids. These two positions, referred to as the Repeat Variable Diresidue (RVD), are highly variable and show a strong correlation with specific nucleotide recognition.
Cytosolic nucleic acid-sensing pathways can enhance immune response to cancer. RIG-I agonist, stem loop RNA (SLR) 14. Tumor growth was significantly delayed and extended survival in mice. SLR14 improved antitumor efficacy of anti-PD1 antibody over single-agent treatment. SLR14 was absorbed by CD11b+ myeloid cells in the tumor microenvironment.
A normal mRNA starts and ends with sections that do not code for amino acids of the actual protein. These sequences at the 5′ and 3′ ends of an mRNA strand are called untranslated regions (UTRs). The two UTRs at their strand ends are essential for the stability of an mRNA and also of a modRNA as well as for the efficiency of translation, i ...
Small activating RNAs (saRNAs) are small double-stranded RNAs that target gene promoters to induce transcriptional gene activation in a process known as RNA activation (RNAa).
A straightforward uptake, like for most small-molecule drugs, is hindered by the polyanionic backbone and the molecular size of ONs. Being adapted from the broad and successful class of Antibody-Drug conjugates , antibodies and antibody analogues are more and more used in research in order to overcome hurdles related to delivery and ...
To limit the rate of off-target cleavage, the therapy uses a highly specific and finely tuned TALEN, which has proven to have little-to-no background off-target interaction. [57] CAR-T immunotherapy is an ex vivo procedure, which means that the patient's immune cells (in this case T-cells ) are extracted and edited using designer nucleases. [ 57 ]
RPN2 is a unique integral glycoprotein in rough ER membrane that is involved in translocation and the maintenance of the structural uniqueness of the rough ER. It is also an essential subunit of N-oligosaccharyl transferase complex that conjugates high mannose oligosaccharides to asparagine residues in the N-X-S/T consensus motif of nascent polypeptide chains.