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16 Ιουλ 2018 · The epigenetic mechanisms through which ncRNAs regulate gene expression involve several RNA types, such as lncRNA and microRNAs (Wei et al., 2017). lncRNAs are molecules over 200 nucleotides in length that regulate the condensation of chromatin, whereas miRNAs are short sequences (21–25 nucleotides) of RNA that modulate RNA silencing and post ...
1 Ιουν 2020 · These genetic results can be applied to generate polygenic risk scores, identify rare causal mutations, evaluate gene by environment interactions, and help determine the specific causes of psychiatric disorders for each individual, all of which can help to guide therapies and treatments.
Herein, we review the evolution of the gene therapy clinical research landscape and describe the gene therapy product development programs evaluated by the FDA in Investigational New Drug applications received in 1988–2019.
16 Νοε 2020 · Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies....
11 Σεπ 2021 · Definition. The term gene therapy refers to the use of different nucleic acids (DNA or RNA) as therapeutic molecules to treat different diseases. It relies on the use of nucleic acid molecules to add a specific gene to a diseased cell (gene augmentation) or to inhibit a specific gene in a diseased cell (gene inhibition).
Of course, the notion of gene therapy has evolved, and in general, we refer to gene therapy when a therapeutic process involves genetic manipulation of the patients' cells with the use of a nucleic acid.
26 Οκτ 2021 · After numerous setbacks at the turn of the century, gene therapy is treating diseases ranging from neuromuscular disorders to cancer to blindness. The success is often qualified, however.
