Delivery of oligonucleotides to bone marrow to modulate ferrochelatase splicing in a mouse model of erythropoietic protoporphyria
Halloy F, Iyer PS, Ćwiek P, Ghidini A, Barman-Aksözen J, Wildner-Verhey van Wijk N, Theocharides APA, Minder EI, Schneider-Yin X, Schümperli D, Hall J. Nucleic Acids Res. 2020 Apr 20. PubMed
Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction
Wang L, Kempton JB, Jiang H, Jodelka FM, Brigande AM, Dumont RA, Rigo F, Lentz JJ, Hastings ML, Brigande JV. Nucleic Acids Res. 2020 Apr 6. PubMed
Directed evolution of adenine base editors with increased activity and therapeutic application
Gaudelli NM, Lam DK, Rees HA, Solá-Esteves NM, Barrera LA, Born DA, Edwards A, Gehrke JM, Lee SJ, Liquori AJ, Murray R, Packer MS, Rinaldi C, Slaymaker IM, Yen J, Young LE, Ciaramella G. Nat Biotechnol. 2020 Apr 13. PubMed
Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity
Yu Y, Leete TC, Born DA, Young L, Barrera LA, Lee SJ, Rees HA, Ciaramella G, Gaudelli NM. Nat Commun. 2020 Apr 28;11(1):2052. PubMed
Imaging small molecule-induced endosomal escape of siRNA
Du Rietz H, Hedlund H, Wilhelmson S, Nordenfelt P, Wittrup A. Nat Commun. 2020 Apr 14;11(1):1809. PubMed
Detailed cellular assessment of albumin-bound oligonucleotides: Increased stability and lower non-specific cell uptake
Lacroix A, Fakih HH, Sleiman HF. J Control Release. 2020 Apr 21. PubMed
Generation and validation of structurally defined antibody-siRNA conjugates
Nanna AR, Kel'in AV, Theile C, Pierson JM, Voo ZX, Garg A, Nair JK, Maier MA, Fitzgerald K, Rader C. Nucleic Acids Res. 2020 Apr 29 PubMed
Big Is Beautiful: Enhanced saRNA Delivery and Immunogenicity by a Higher Molecular Weight, Bioreducible, Cationic Polymer
Blakney AK, Zhu Y, McKay PF, Bouton CR, Yeow J, Tang J, Hu K, Samnuan K, Grigsby CL, Shattock RJ, Stevens MM. ACS Nano. 2020 Apr 20. PubMed
Cationic Amphiphilic Drugs Boost the Lysosomal Escape of Small Nucleic Acid Therapeutics in a Nanocarrier-Dependent Manner
Van de Vyver T, Bogaert B, De Backer L, Joris F, Guagliardo R, Van Hoeck J, Merckx P, Van Calenbergh S, Ramishetti S, Peer D, Remaut K, De Smedt SC, Raemdonck K. ACS Nano. 2020 Apr 28;14(4):4774-4791. PubMed
Vaccination against non-mutated neoantigens induced in recurrent and future tumors
Garrido G, Schrand B, Levay A, Rabasa Capote A, Ferrantella A, Da Silva DM, D'Eramo F, Marijt KA, Zhang Z, Kwon D, Kortylewski M, Kast WM, Dudeja V, van Hall T, Gilboa E. Cancer Immunol Res. 2020 Apr 15. PubMed
