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Of the four types, mRNA-based therapy is the only type which is based on triggering synthesis of proteins within cells, making it particularly useful in vaccine development. [3] Antisense RNA is complementary to coding mRNA and is used to trigger mRNA inactivation to prevent the mRNA from being used in protein translation. [4]
Mechanism of self-amplifying mRNA (saRNA) used for antigen production. The ORF encoding the antigen can also be substituted with a protein for use in protein replacement therapy. This sequence encodes the protein of interest, used as an antigen in the case of vaccines or for protein replacement therapies. The gene of interest replaces the viral ...
This strategy is referred to as gene replacement therapy and could be employed to treat inherited retinal diseases. [17] [66] While the concept of gene replacement therapy is mostly suitable for recessive diseases, novel strategies have been suggested that are capable of also treating conditions with a dominant pattern of inheritance.
Our first CDP mRNA therapy targets atherosclerosis and, as with all mRNA-based approaches, delivery is essential to achieving therapeutic benefit. We are excited to be working with Genevant, a recognized leader in the LNP space,” said Reason, Chief Executive Officer and co-founder of Repair.
GAITHERSBURG, Md., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today announced that the first patient has been dosed in its first-in-human Phase 1 trial of Descartes-15, the Company’s next ...
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 ...
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