过去一年中添加的文章,按日期排序

Development of Organ Targeting Lipid Nanoparticles with Low Immunogenicity and Its Application in the Treatment of Pulmonary Fibrosis

M Cheng, X Yu, S Qi, K Yang, M Lu… - … (International ed. in … - pubmed.ncbi.nlm.nih.gov
2 天前 - non-viral delivery system, lipid nanoparticles (LNPs) were approved by the FDA,
propelling the advancements of gene therapy… synergy of gene therapy and drug therapy. This …

Creation of an in vitro model of GM1 gangliosidosis by CRISPR/Cas9 knocking-out the GLB1 gene in SH-SY5Y human neuronal cell line

K Hosseini, J Fallahi, H Aligholi… - Cell biochemistry …, 2024 - pubmed.ncbi.nlm.nih.gov
3 天前 - genes, and then the top correlated genes were tested for further evaluation of
knock-out effects. The nonviral … exons 14 and 16 of the GLB1 gene into SH-SY5Y cells led to the …

Advancements of Gene Therapy in Cancer Treatment: A Comprehensive Review

MDM Abas, MFM Asri, NAS Yusafawi… - … -Research and Practice, 2024 - Elsevier
3 天前 - … of gene therapies can generally be classified into two methods: viral and non-viral
are also known as transduction technologies while non-viral vectors can be done by either …

[HTML][HTML] Potential Targeting Mechanisms for Bone-Directed Therapies

B Celik, AF Leal, S Tomatsu - International Journal of Molecular Sciences, 2024 - mdpi.com
3 天前 - gene delivery (eg, direct infusion of viral or non-viral vectors with no modifications
on capsid, envelope, or nanoparticles), and cell therapy … The preclinical gene therapy

Characterization and Biochemical Study of Programmed Ribosomal Frameshifting in Human and Viral mRNAs

X Zhou - opensiuc.lib.siu.edu
3 天前 - … Moreover, in more than half of these cases, the frameshift site locates far upstream
from the stop codon of the 0 reading frame, which is nonviral-like. Using dual luciferase …

[HTML][HTML] Minicircle DNA vectors: A breakthrough in non-viral delivery of CRISPR base editors?

M Kanada, AA Gilad - Molecular Therapy-Nucleic Acids, 2024 - cell.com
4 天前 - gene therapy technologies. These include the use of viral vectors to deliver functional
genes… and hemophilia, and the development of targeted gene-editing tools like zinc finger …

Adeno-Associated Virus Engineering and Load Strategy for Tropism Modification, Immune Evasion and Enhanced Transgene Expression

X Zhou, J Liu, S Xiao, X Liang, Y Li, F Mo… - International Journal …, 2024 - Taylor & Francis
4 天前 - … 14 gene therapy products. With the increasing interest in gene therapy, feasible
gene … delivery vectors can be divided into viral and non-viral delivery vectors. For example, …

Exploring non-viral methods for the delivery of CRISPR-Cas ribonucleoprotein to hematopoietic stem cells

Z Molaei, Z Jabbarpour, A Omidkhoda… - … Research & Therapy, 2024 - Springer
4 天前 - non-viral delivery systems for gene editing of HSCs could significantly enhance the
therapeutic … guidance for researchers working on gene therapy applications involving HSCs. …

Nanomaterial Translational Nanomedicine for Anti‐HIV and Anti‐bacterial

H Luo, Y Song - Nanomedicine: Fundamentals, Synthesis, and …, 2024 - Wiley Online Library
7 天前 - … The application progresses of nanomedicines, which includes nanoantitumor
drugs, nanopeptide–protein drugs, and nanoformulations of nonviral vector gene drugs, are …

[HTML][HTML] Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress

Y Liu, X Zong, W Cao, W Zhang, N Zhang, N Yang - Biomolecules, 2024 - mdpi.com
8 天前 - … research, especially in gene therapy, are significantly … of new compounds,
gene-editing techniques, and gene loci offering … viral or non-viral vectors to correct genetic defects …