Current state of human gene therapy: approved products and vectors

AY Shchaslyvyi, SV Antonenko, MG Tesliuk… - Pharmaceuticals, 2023 - mdpi.com
In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking
identification of the first recombinant DNA in 1972. This achievement set the stage for future …

Advancing nucleic acid delivery through cationic polymer design: non-cationic building blocks from the toolbox

I Porello, N Bono, G Candiani, F Cellesi - Polymer Chemistry, 2024 - pubs.rsc.org
Polymers used for the delivery of nucleic acids (NAs) typically possess ionizable, cationic
moieties enabling their electrostatic interactions with negatively charged NAs and form …

pH-Responsive Micelles Containing Quinine Functionalities Enhance Intracellular Gene Delivery and Expression

NW Kreofsky, P Roy, TM Reineke - Bioconjugate Chemistry, 2024 - ACS Publications
Quinine is a promising building block for creating polymer carriers for intracellular nucleic
acid delivery. This is due to its ability to bind to genetic material through intercalation and …

Blended block polycation micelles enhance antisense oligonucleotide delivery

MG Hanson, CJ Grimme, NW Kreofsky… - Bioconjugate …, 2023 - ACS Publications
Nucleic acid-based medicines and vaccines are becoming an important part of our
therapeutic toolbox. One key genetic medicine is antisense oligonucleotides (ASOs), which …

Cationic Micelles Outperform Linear Polymers for Delivery of Antisense Oligonucleotides in Serum: An Exploration of Polymer Architecture, Cationic Moieties, and Cell …

MG Hanson, CJ Grimme, CF Santa Chalarca… - Bioconjugate …, 2022 - ACS Publications
Antisense oligonucleotides (ASOs) are an important emerging therapeutic; however, they
struggle to enter cells without a delivery vehicle, such as a cationic polymer. To understand …

Enhanced ASO-Mediated Gene Silencing with Lipophilic PH-Responsive Micelles

CJ Grimme, MG Hanson, TM Reineke - Bioconjugate Chemistry, 2023 - ACS Publications
Herein, we examine the ASO-mediated gene silencing efficiency of pH-responsive micelles,
by incorporating 2-(diisopropylamino) ethyl methacrylate (DIP) into the micelle core and …

Kinetically Controlled Polyelectrolyte Complex Assembly of microRNA‐Peptide Nanoparticles toward Treating Mesothelioma

CF Anderson, A Singh, T Stephens… - Advanced …, 2024 - Wiley Online Library
Broad size distributions and poor long‐term colloidal stability of microRNA‐carrying
nanoparticles, especially those formed by polyelectrolyte complexation, represent major …

Nanomedicine-RNAi interface: Architypes in prostate cancer therapeutics

S Shah, P Famta, DA Srinivasarao, S Shahrukh… - Journal of Drug Delivery …, 2024 - Elsevier
Prostate cancer (PCa) is the most frequent cancer among men throughout the world. The
available conventional therapies such as androgen deprivation therapy and surgical …

Molecular Dynamics Simulations Elucidate the Molecular Organization of Poly (beta-amino ester) Based Polyplexes for siRNA Delivery

KM Steinegger, L Allmendinger, S Sturm… - Nano Letters, 2024 - ACS Publications
Cationic polymers are known to efficiently deliver nucleic acids to target cells by
encapsulating the cargo into nanoparticles. However, the molecular organization of these …

Beyond Lipids: Exploring Advances in Polymeric Gene Delivery in the Lipid Nanoparticles Era

CM Jogdeo, K Siddhanta, A Das, L Ding… - Advanced …, 2024 - Wiley Online Library
The recent success of gene therapy during the COVID‐19 pandemic has underscored the
importance of effective and safe delivery systems. Complementing lipid‐based delivery …