Genomics in Toxicology DOI
David Scoville, Christopher M. Schaupp, Terrance J. Kavanagh

и другие.

Elsevier eBooks, Год журнала: 2024, Номер unknown

Опубликована: Янв. 1, 2024

Язык: Английский

Scaffolds of Chitosan-metallic hybrids as antimicrobial wound dressing DOI
Shengyu Zhang, Muhammad Ali,

Farooq Nawaz

и другие.

Journal of Molecular Liquids, Год журнала: 2024, Номер unknown, С. 126541 - 126541

Опубликована: Ноя. 1, 2024

Язык: Английский

Процитировано

4

Polymeric nanocarriers for gene delivery DOI Creative Commons
Jiayuan Zhang, Xinyu Yang, Zhenyu Chang

и другие.

Asian Journal of Pharmaceutical Sciences, Год журнала: 2025, Номер 20(1), С. 101015 - 101015

Опубликована: Янв. 5, 2025

The recent commercialization of gene products has sparked significant interest in therapy, necessitating efficient and precise delivery via various vectors. Currently, viral vectors lipid-based nanocarriers are the predominant choices have been extensively investigated reviewed. Beyond these vectors, polymeric also hold promise therapeutic owing to their versatile functionalities, such as improving stability, cellar uptake endosomal escape nucleic acid drugs, along with targeted tissues. This review presents a brief overview status quo emerging for delivery, focusing on key cationic polymers, nanocarrier types, preparation methods. It highlights diseases, strategies improve efficiency, potential future directions this research area. is hoped inspire development, optimization, clinical translation highly delivery.

Язык: Английский

Процитировано

0

Gene Therapy for Cystic Fibrosis: Overcoming Current Limitations and Future Directions DOI

Nadir Ali Wassan,

Asfia Qammar,

Sarah Bürki

и другие.

Indus journal of bioscience research., Год журнала: 2025, Номер 3(3), С. 133 - 145

Опубликована: Март 16, 2025

Cystic fibrosis (CF) is a lethal inherited disorder resulting from mutations in the CFTR gene that cause improper chloride ion movement and clogging of lungs with mucus. While classic therapies have been augmented by modulators airway clearance techniques, these remain limited expense, specificity for mutation, need lifetime therapy. Gene therapy has suggested as curative treatment, wherein goal to repair defective through techniques such CRISPR-Cas9, viral/non-viral vectors, stem cell-based This study employed mixed-methods setting, integrating survey-based assessment meta-analysis existing literature evaluate awareness, perception, barriers implementation among CF patients caregivers Punjab, Pakistan. A questionnaire was given 65 participants, qualitative data analysis SPSS established significant awareness gap, where socioeconomic status education level were major determinants. Chi-square ANOVA tests statistically relationships between demographic variables acceptance. Moreover, found consistent trends current research indicating similar problems accessibility, ethical concerns, necessity improved delivery systems. The results reaffirm further patient education, increased affordability, more clinical verification make therapeutic reality. In general, underscores promise while encouraging multidisciplinary methods overcome limitations. examines perceptions cystic Pakistan, revealing low knowledge levels influenced factors. Challenges include cost, safety implications. Future efforts must integrate research, policy reforms, advocacy enhance therapy’s feasibility availability.

Язык: Английский

Процитировано

0

Non-viral mRNA delivery to the lungs DOI Creative Commons

Lauren Healy,

B. Seto,

Haissi Cui

и другие.

Biomaterials Science, Год журнала: 2025, Номер unknown

Опубликована: Янв. 1, 2025

This review highlights emerging strategies for lung-targeted mRNA delivery using non-viral vehicles, describing advances in bioinspired design and nanoparticle engineering.

Язык: Английский

Процитировано

0

Synthesis of ionizable lipopolymers using split-Ugi reaction for pulmonary delivery of various size RNAs and gene editing DOI Creative Commons
Kseniya Yu. Vlasova, Alexander M. Kerr, Nathan D. Pennock

и другие.

Nature Communications, Год журнала: 2025, Номер 16(1)

Опубликована: Апрель 29, 2025

We present an efficient method for synthesizing cationic poly(ethylene imine) derivatives using the multicomponent split-Ugi reaction to create a library of functional ionizable lipopolymers. Here we show 155 polymers, formulated into polyplexes, establish structure-activity relationships essential endosomal escape and transfection. A lead structure is identified, lipopolymer-lipid hybrid nanoparticles are developed deliver mRNA lung endothelium immune cells, including T with low in vivo toxicity. These significant improvements delivery compared vivo-JetPEI® demonstrate effective therapeutic mRNA(s) various sizes. IL-12 mRNA-loaded delay Lewis Lung cancer progression, while human CFTR restores protein function knockout mice. Additionally, CRISPR-Cas9 delivery, achieving gene editing tissue successful PD-1 cells results highlight platform's potential systemic therapy delivery.

Язык: Английский

Процитировано

0

Tailoring diblock copolymers for efficient siPLK1 delivery and enhanced gene therapy of orthotopic osteosarcoma DOI
Hongyang Zhao, Chenglin Zhang, Chang Tian

и другие.

Journal of Materials Chemistry B, Год журнала: 2025, Номер unknown

Опубликована: Янв. 1, 2025

The structure of the engineered polymer carrier BP 20 and its complexation with siRNA. Schematic illustration /siPLK1 nanoparticle (NP) mediated cellular internalization PLK1 gene silencing in osteosarcoma (143B) cells.

Язык: Английский

Процитировано

0

Synthesis of ionizable lipopolymers using split-Ugi reaction for pulmonary delivery of various size RNAs and gene editing DOI Open Access
Kseniya Yu. Vlasova, Alexander M. Kerr, Nathan D. Pennock

и другие.

bioRxiv (Cold Spring Harbor Laboratory), Год журнала: 2024, Номер unknown

Опубликована: Июнь 13, 2024

We present an efficient approach for synthesizing cationic poly(ethylene imine) derivatives using the multicomponent split-Ugi reaction to rapidly create a library of complex functional ionizable lipopolymers. synthesized diverse 155 polymers, formulated them into polyplexes establish structure-activity relationships crucial endosomal escape and transfection. After discovering lead structure, lipopolymer-lipid hybrid nanoparticles are introduced preferentially deliver elicit effective mRNA transfection in lung endothelium immune cells, including T cells with low

Язык: Английский

Процитировано

2

Genomics in Toxicology DOI
David Scoville, Christopher M. Schaupp, Terrance J. Kavanagh

и другие.

Elsevier eBooks, Год журнала: 2024, Номер unknown

Опубликована: Янв. 1, 2024

Язык: Английский

Процитировано

0