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Nonviral Gene Delivery with Cationic Glycopolymers
Craig Van Bruggen1, Joseph K Hexum1, Zhe Tan1
1Department of Chemistry , University of Minnesota , 207 Pleasant Street SE , Minneapolis , Minnesota 55455 , United States.
Accounts of Chemical Research
|April 18, 2019
Summary
Carbohydrate-based polymers, or glycopolymers, offer efficient and biocompatible nonviral gene delivery. These novel materials enhance nucleic acid delivery by improving degradation, targeting, and stability, paving the way for safer gene therapies.
Area of Science:
- Biomaterials Science
- Polymer Chemistry
- Gene Therapy Delivery
Background:
- Current gene therapies often use viral vectors, but nonviral methods like polymer-based systems are sought for their cost-effectiveness and tunability.
- Existing polymeric transfection reagents face challenges with efficiency and toxicity, limiting their clinical application.
- Carbohydrates can enhance the biocompatibility of synthetic polymers for improved gene delivery.
Purpose of the Study:
- To develop and characterize novel carbohydrate-based cationic polymers (glycopolymers) for enhanced nonviral gene delivery.
- To investigate the structure-activity relationships of these glycopolymers for improved efficiency and biocompatibility.
- To explore the potential of glycopolymers in delivering nucleic acid cargo for gene therapy and gene editing.
Main Methods:
- Synthesis of poly(glycoamidoamine)s (PGAAs) via step-growth polymerization of monosaccharides and ethyleneamines.
- Functionalization of monosaccharides as vinyl monomers for diblock copolymer synthesis using radical addition-fragmentation chain-transfer (RAFT) polymerization.
- Evaluation of polymer-DNA complexation, colloidal stability, in vitro cellular uptake, in vivo tissue targeting, and stability upon lyophilization.
Main Results:
- PGAAs demonstrated efficient and biocompatible gene transfection.
- Glucose-containing diblock copolymers provided colloidal stability comparable to PEG.
- N-acetyl-d-galactosamine variants enhanced receptor-mediated uptake by hepatocytes in vitro and liver targeting in vivo.
- Trehalose-based copolymers improved colloidal stability and resistance to aggregation after lyophilization.
Conclusions:
- Glycopolymers effectively deliver nucleic acid cargo into various human cell types.
- Carbohydrate incorporation enhances vehicle degradation, tissue targeting, colloidal stabilization, and lyophilization stability.
- These advancements in glycopolymers hold promise for the translation of safe and efficient gene therapy and gene editing applications.
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