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A nucleoside-modified messenger RNA (modRNA) is a synthetic messenger RNA (mRNA) in which some nucleosides are replaced by other naturally modified nucleosides or by synthetic nucleoside analogues.
Molecular medicine is a broad field, where physical, chemical, biological, bioinformatics and medical techniques are used to describe molecular structures and mechanisms, identify fundamental molecular and genetic errors of disease, and to develop molecular interventions to correct them. [1]
Gene therapy was first conceptualized in the 1960s, when the feasibility of adding new genetic functions to mammalian cells began to be researched. Several methods to do so were tested, including injecting genes with a micropipette directly into a living mammalian cell, and exposing cells to a precipitate of DNA that
These models are the most accurate in vitro models available to researchers and facilitate the development of personalized drugs and diagnostics, particularly in oncology. [32] Gene targeting has also been investigated for gene therapy to correct disease-causing mutations. However the low efficiency of delivery of the gene-targeting machinery ...
RNA aptamers are not so much “created” as “selected.” To develop an RNA aptamer capable of selective binding to a molecular target, a method known as Systematic Evolution of Ligands by EXponential Enrichment (SELEX) is used to isolate a unique RNA aptamer from a pool of ~10^13 to 10^16 different aptamers, otherwise known as a library.
Immune gene therapy is a targeted approach to cancer therapy where actual immune cells of the patient and their genes are manipulated to produce an anti-tumor response. [5] The body's own immune system is used to attack the tumor cells, therefore the immune system can naturally attack the specific cancer cells again to in the future if ...
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 .
Viral vector manufacturing methods often vary by vector, although most utilize an adherent or suspension-based system with mammalian cells. [72] For viral vector production on a smaller, laboratory setting, static cell culture systems like Petri dishes are typically used.