Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals January 9, 2017
Reported February 19, 2017 as A new way Forward for Gene Therapy
If you want an explanation of how mRNA works within a cell, check out the video above from the Khan Academy.
Unfortunately, the speaker seems to think that sickle-cell disease is caused by a mutation. All serious scientists know that the hemoglobin S variant is a biophysically constrained nutrient energy-dependent ecological adaptation that protects populations from virus-driven genomic entropy due to phages in the class of Sporazoa, which includes the parasite that causes malaria.
See also: HbVar: A Database of Human Hemoglobin Variants and Thalassemias
More than 1200 hemoglobin variants have been reported.
Here is more information on the known variants Dobzhansky (1964) reported that “Ingram and others found that hemoglobin S differs from A in the substitution of just a single amino acid, valine in place of glutamic acid in the beta chain of the hemoglobin molecule.”
If you want details about how energy-dependent changes in the microRNA/messenger balance link amino acid substitutions in supercoiled DNA to all healthy longevity compared to the fact that virus-driven energy theft is linked from mutations to all pathology, see:
Energy as information and constrained endogenous RNA interference
Feedback loops link quantized energy as information to biophysically constrained RNA-mediated protein folding chemistry. Light induced energy-dependent changes link angstroms to ecosystems from classical physics to chemistry/chirality and to molecular epigenetics/autophagy.
The National Microbiome Initiative links microbial quorum sensing to the physiology of reproduction via endogenous RNA interference and chromosomal rearrangements. The rearrangements link energy-dependent fixed amino acid substitutions to the Precision Medicine Initiative via genome wide inferences of natural selection.
This detailed representation of energy-dependent natural selection for codon optimality links biologically- based cause and effect from G protein-coupled receptors to RNA-mediated amino acid substitutions and the functional structure of supercoiled DNA. Energy-dependent polycombic ecological adaptations are manifested in supercoiled DNA. Chromosomal inheritance links the adaptations from morphological phenotypes to healthy longevity via behavioral phenotypes.
For contrast, virus-driven energy theft is the link from messenger RNA degradation to negative supercoiling, constraint breaking mutations, and hecatombic evolution. The viral hecatomb links transgenerational epigenetic inheritance from archaea to Zika virus-damaged DNA, which typically is repaired by endogenous RNA interference and fixation of RNA-mediated amino acid substitutions in organized genomes
The papers by Jaeger (microbiology) and Noble (physiology) are strong steps forward in this regard. This is perhaps the area where most needs to be done, across the board in all domains. Areas where striking progress is being made in this regard are epigenetics  and social neuroscience . These papers are in fact dealing with top-down causation: the way they provide experimental confirmation of this concept needs to be made more explicit.
What don’t pseudoscientists understand about the need to link energy-dependent RNA-mediated protein folding chemistry from metabolism in microbes to their nutrient-dependent pheromone-controlled physiology of energy-dependent reproduction before inventing more theories based on definitions and assumptions about interpretations of data?
Each example of RNA editing will continue to link chirality to autophagy and link endogenous RNA interference (RNA editing) to supercoiled DNA via amino acids in supercoiled DNA. If example of virus-driven energy theft are not sufficient to show that it is the cause of all pathology, someone will need to show what causes the mutations that are linked to all pathology.
See also: Genomic Energy Landscapes
Their perspective on the energy landscape theory asks the same old questions about chromosomal inheritance and provides no basis for linking experimental evidence of top-down energy-dependent causation from RNA-mediated protein folding chemistry to all biodiversity via the physiology of pheromone-controlled reproduction. It is another refutation of theistic evolution because it fails to link anything to anything else.
See for comparison: Coevolutionary information, protein folding landscapes, and the thermodynamics of natural selection (2014)
Comparing the details of the physical and information theoretic models has already suggested ways of improving the prediction of mutational effects on the stability of protein sequence/structure pairs.
Virus-driven energy theft causes mutations, which link messenger RNA degradation to genomic entropy in hosts because the energy is used for amino acid substitutions that stabilize the organized genomes of viruses. How much evidence of virus-driven energy theft and pathology can be viewed in the context of the fossil record compared to the frozen corpse of “the Tyrolean Iceman?”
See: miRNAs in ancient tissue specimens of the Tyrolean Iceman reported as: Otzi the Iceman: Researchers validate the stability of genetic markers
…microRNAs can remain stable even after 5,300 years.
From the same first author: The missense of smell: functional variability in the human odorant receptor repertoire
The variability of single odorant receptors is clearly linked from energy-dependent changes in base pairs to the microRNA/messenger RNA balance. The balance links SNPs to natural selection for energy-dependent codon optimality.
Codon optimality links single amino acid substitutions from RNA editing to nutrient-dependent pheromone-controlled adaptations. That fact links natural selection from ecological variations to ecological adaptations. The energy-dependent adaptations obviously occur in species from microbes (including viruses) to man. If the adaptations did not occur in all living genera, the microRNAs could not have been linked to genetic markers in the ancient tissue specimens via RNA editing.
See also: Contribution of epigenetic mechanisms to variation in cancer risk among tissues
…the risk of cancer in any given tissue would be correlated with the number of abnormally methylated precancer cells in that cell type. Because de novo modification appears to take place almost exclusively on CpG islands that are already silenced by polycomb in the normal tissue (8), we suggest that this modification works by preventing these genes from becoming activated, thereby inhibiting normal tissue differentiation, causing clonal selection for cells that may predispose to cancer (31). Indeed, many of these methylation targets have been shown to be “driver” genes in a number of different cell types (Fig. S6).
Once again we see that serious scientists know the difference between energy-dependent polycombic ecological adaptations and the virus-driven hecatombic evolution of all pathology, but they clearly do not know how to express what they know in terms that are meaningful to those who know how to link angstroms to ecosystems in all living genera.