Non-viral vectors for gene-based therapy
Gene-based therapy is the intentional modulation of gene expression in specific cells to treat
pathological conditions. This modulation is accomplished by introducing exogenous nucleic …
pathological conditions. This modulation is accomplished by introducing exogenous nucleic …
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
JA Zuris, DB Thompson, Y Shu, JP Guilinger… - Nature …, 2015 - nature.com
Efficient intracellular delivery of proteins is needed to fully realize the potential of protein
therapeutics. Current methods of protein delivery commonly suffer from low tolerance for …
therapeutics. Current methods of protein delivery commonly suffer from low tolerance for …
RNA-based diagnostic and therapeutic strategies for cardiovascular disease
D Lu, T Thum - Nature Reviews Cardiology, 2019 - nature.com
Cardiovascular diseases are the leading cause of death globally and are associated with
increasing financial expenditure. With the availability of next-generation sequencing …
increasing financial expenditure. With the availability of next-generation sequencing …
Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs
KJ Kauffman, JR Dorkin, JH Yang, MW Heartlein… - Nano …, 2015 - ACS Publications
Intracellular delivery of messenger RNA (mRNA) has the potential to induce protein
production for many therapeutic applications. Although lipid nanoparticles have shown …
production for many therapeutic applications. Although lipid nanoparticles have shown …
Pathophysiology of cardiac hypertrophy and heart failure: signaling pathways and novel therapeutic targets
The onset of heart failure is typically preceded by cardiac hypertrophy, a response of the
heart to increased workload, a cardiac insult such as a heart attack or genetic mutation …
heart to increased workload, a cardiac insult such as a heart attack or genetic mutation …
N1-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced …
O Andries, S Mc Cafferty, SC De Smedt, R Weiss… - Journal of Controlled …, 2015 - Elsevier
Messenger RNA as a therapeutic modality is becoming increasingly popular in the field of
gene therapy. The realization that nucleobase modifications can greatly enhance the …
gene therapy. The realization that nucleobase modifications can greatly enhance the …
Exosome-mediated mRNA delivery in vivo is safe and can be used to induce SARS-CoV-2 immunity
Functional delivery of mRNA has high clinical potential. Previous studies established that
mRNAs can be delivered to cells in vitro and in vivo via RNA-loaded lipid nanoparticles …
mRNAs can be delivered to cells in vitro and in vivo via RNA-loaded lipid nanoparticles …
Efficient targeting and activation of antigen-presenting cells in vivo after modified mRNA vaccine administration in rhesus macaques
mRNA vaccines are rapidly emerging as a powerful platform for infectious diseases because
they are well tolerated, immunogenic, and scalable and are built on precise but adaptable …
they are well tolerated, immunogenic, and scalable and are built on precise but adaptable …
Pkm2 regulates cardiomyocyte cell cycle and promotes cardiac regeneration
Background: The adult mammalian heart has limited regenerative capacity, mostly
attributable to postnatal cardiomyocyte cell cycle arrest. In the last 2 decades, numerous …
attributable to postnatal cardiomyocyte cell cycle arrest. In the last 2 decades, numerous …
Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals
A Thess, S Grund, BL Mui, MJ Hope, P Baumhof… - Molecular Therapy, 2015 - cell.com
Being a transient carrier of genetic information, mRNA could be a versatile, flexible, and safe
means for protein therapies. While recent findings highlight the enormous therapeutic …
means for protein therapies. While recent findings highlight the enormous therapeutic …