We are analyzing https://link.springer.com/article/10.1186/s13073-017-0450-0.

Title:
Advances in the delivery of RNA therapeutics: from concept to clinical reality | Genome Medicine
Description:
The rapid expansion of the available genomic data continues to greatly impact biomedical science and medicine. Fulfilling the clinical potential of genetic discoveries requires the development of therapeutics that can specifically modulate the expression of disease-relevant genes. RNA-based drugs, including short interfering RNAs and antisense oligonucleotides, are particularly promising examples of this newer class of biologics. For over two decades, researchers have been trying to overcome major challenges for utilizing such RNAs in a therapeutic context, including intracellular delivery, stability, and immune response activation. This research is finally beginning to bear fruit as the first RNA drugs gain FDA approval and more advance to the final phases of clinical trials. Furthermore, the recent advent of CRISPR, an RNA-guided gene-editing technology, as well as new strides in the delivery of messenger RNA transcribed in vitro, have triggered a major expansion of the RNA-therapeutics field. In this review, we discuss the challenges for clinical translation of RNA-based therapeutics, with an emphasis on recent advances in delivery technologies, and present an overview of the applications of RNA-based drugs for modulation of gene/protein expression and genome editing that are currently being investigated both in the laboratory as well as in the clinic.
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Keywords {🔍}

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Functional genomics and rheumatoid arthritis: where have we been and where should we go?

Schema {🗺️}

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         headline:Advances in the delivery of RNA therapeutics: from concept to clinical reality
         description:The rapid expansion of the available genomic data continues to greatly impact biomedical science and medicine. Fulfilling the clinical potential of genetic discoveries requires the development of therapeutics that can specifically modulate the expression of disease-relevant genes. RNA-based drugs, including short interfering RNAs and antisense oligonucleotides, are particularly promising examples of this newer class of biologics. For over two decades, researchers have been trying to overcome major challenges for utilizing such RNAs in a therapeutic context, including intracellular delivery, stability, and immune response activation. This research is finally beginning to bear fruit as the first RNA drugs gain FDA approval and more advance to the final phases of clinical trials. Furthermore, the recent advent of CRISPR, an RNA-guided gene-editing technology, as well as new strides in the delivery of messenger RNA transcribed in vitro, have triggered a major expansion of the RNA-therapeutics field. In this review, we discuss the challenges for clinical translation of RNA-based therapeutics, with an emphasis on recent advances in delivery technologies, and present an overview of the applications of RNA-based drugs for modulation of gene/protein expression and genome editing that are currently being investigated both in the laboratory as well as in the clinic.
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      headline:Advances in the delivery of RNA therapeutics: from concept to clinical reality
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         Clinical trial
         CRISPR
         Gene editing
         Gene therapy
         Messenger RNA delivery
         mRNA vaccine
         RNA nanoparticle
         Short interfering RNA delivery
         Human Genetics
         Metabolomics
         Bioinformatics
         Medicine/Public Health
         general
         Cancer Research
         Systems Biology
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            address:
               name:David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, USA
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               type:PostalAddress
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      name:Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, USA
      name:Harvard and MIT Division of Health Science and Technology, Massachusetts Institute of Technology, Cambridge, USA

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