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https://www.nature.com/articles/s41467-018-04894-2
Jun 26, 2018 · Here we demonstrate that in utero delivery of PLGA nanoparticles loaded with PNA/DNA is a safe and effective means of achieving clinically relevant frequencies of site-specific, non-enzymatic gene editing in a mammalian fetus that results in sustained postnatal alleviation of disease.Author: Adele S. Ricciardi, Raman Bahal, Raman Bahal, James S. Farrelly, Elias Quijano, Anthony H. Bianchi, ...
https://www.researchgate.net/publication/325997823_In_utero_nanoparticle_delivery_for_site-specific_genome_editing
In utero introduction of nanoparticles containing peptide nucleic acids (PNAs) and donor DNAs corrects a disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia, yielding sustained postnatal elevation of blood hemoglobin levels into the normal range,...
http://adsabs.harvard.edu/abs/2018NatCo...9.2481R
In utero introduction of nanoparticles containing peptide nucleic acids (PNAs) and donor DNAs corrects a disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia, yielding sustained postnatal elevation of blood hemoglobin levels into the normal range, reduced reticulocyte counts, reversal of splenomegaly, and improved survival, with no detected off-target mutations in …
https://www.youtube.com/watch?v=i7T8ctqoftI
Jun 26, 2018 · In utero introduction of nanoparticles containing peptide nucleic acids (PNAs) and donor DNAs corrects a disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia ...
https://www.altmetric.com/details/44149944
In utero introduction of nanoparticles containing peptide nucleic acids (PNAs) and donor DNAs corrects a disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia, yielding sustained postnatal elevation of blood hemoglobin levels into the normal range, reduced reticulocyte counts, reversal of splenomegaly, and improved survival, with no detected off-target mutations in …
https://www.sciencedaily.com/releases/2018/07/180709120133.htm
Jul 09, 2018 · Their technique uses an FDA-approved nanoparticle to deliver PNA molecules paired with donor DNA to the site of a genetic mutation. ... In utero nanoparticle delivery for site-specific genome ...
https://scitechdaily.com/nanoparticles-cure-blood-disorder-provide-site-specific-gene-editing/
With the combined efforts of three Yale laboratories, researchers conducted the first demonstration of site-specific gene editing in a fetus, correcting a mutation that causes a severe form of anemia.
https://www.genengnews.com/topics/omics/fetal-gene-editing-with-synthetic-nucleotides-cures-beta-thalassemia-in-mice/
Jul 10, 2018 · “Here we demonstrate that in utero delivery of PLGA nanoparticles loaded with PNA/DNA is a safe and effective means of achieving clinically relevant frequencies of site-specific, nonenzymatic gene...
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