Cas13b-mediated RNA targeted therapy alleviates genetic dilated cardiomyopathy in mice

[1] Bongianino R(2017)Allele-specific silencing of mutant mRNA rescues ultrastructural and arrhythmic phenotype in mice carriers of the R4496C mutation in the ryanodine receptor gene (RYR2) Circul Res 121 525-36

[2] Denegri M(2018)Hypoxia-inducible factor 2-alpha-dependent induction of amphiregulin dampens myocardial ischemia-reperfusion injury Nat Commun 9 816-88

[3] Mazzanti A(2016)Designing idiosyncratic hmPCL-siRNA nanoformulated capsules for silencing and cancer therapy Nanomedicine: Nanatechnol Biology Med 12 579-6

[4] Lodola F(2014)Liposomal siRNA nanocarriers for cancer therapy Adv Drug Deliv Rev 66 110-e7

[5] Vollero A(2017)Lipophilic siRNA targets albumin in situ and promotes bioavailability, Tumor penetration, and carrier-free gene silencing Proc Natl Acad Sci USA 114 E6490-94

[6] Boncompagni S(2019)A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system Nat Biotechnol 37 884-33

[7] Koeppen M(2018)Systemic delivery of BACE1 siRNA through neuron-targeted nanocomplexes for treatment of Alzheimer’s Disease J Controlled Release: Official J Controlled Release Soc 279 220-75

[8] Lee JW(2018)Nanotechnology-based strategies for siRNA brain delivery for Disease Therapy Trends Biotechnol 36 562-44

[9] Seo SW(2016)Clustering siRNA conjugates for MMP-responsive therapeutics in chronic wounds of diabetic animals Nanoscale 8 13236-26

[10] Brodsky KS(2017)Efficiency and safety of β-CD-(D(3))(7) as siRNA carrier for decreasing Matrix Metalloproteinase-9 expression and improving Wound Healing in Diabetic rats ACS Appl Mater Interfaces 9 17417-75

[1] Bongianino R(2017)Allele-specific silencing of mutant mRNA rescues ultrastructural and arrhythmic phenotype in mice carriers of the R4496C mutation in the ryanodine receptor gene (RYR2) Circul Res 121 525-36

[2] Denegri M(2018)Hypoxia-inducible factor 2-alpha-dependent induction of amphiregulin dampens myocardial ischemia-reperfusion injury Nat Commun 9 816-88

[3] Mazzanti A(2016)Designing idiosyncratic hmPCL-siRNA nanoformulated capsules for silencing and cancer therapy Nanomedicine: Nanatechnol Biology Med 12 579-6

[4] Lodola F(2014)Liposomal siRNA nanocarriers for cancer therapy Adv Drug Deliv Rev 66 110-e7

[5] Vollero A(2017)Lipophilic siRNA targets albumin in situ and promotes bioavailability, Tumor penetration, and carrier-free gene silencing Proc Natl Acad Sci USA 114 E6490-94

[6] Boncompagni S(2019)A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system Nat Biotechnol 37 884-33

[7] Koeppen M(2018)Systemic delivery of BACE1 siRNA through neuron-targeted nanocomplexes for treatment of Alzheimer’s Disease J Controlled Release: Official J Controlled Release Soc 279 220-75

[8] Lee JW(2018)Nanotechnology-based strategies for siRNA brain delivery for Disease Therapy Trends Biotechnol 36 562-44

[9] Seo SW(2016)Clustering siRNA conjugates for MMP-responsive therapeutics in chronic wounds of diabetic animals Nanoscale 8 13236-26

[10] Brodsky KS(2017)Efficiency and safety of β-CD-(D(3))(7) as siRNA carrier for decreasing Matrix Metalloproteinase-9 expression and improving Wound Healing in Diabetic rats ACS Appl Mater Interfaces 9 17417-75