Various AAV serotypes and their applications in gene therapy: an overview

SS Issa, AA Shaimardanova, VV Solovyeva… - Cells, 2023 - mdpi.com
Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

[HTML][HTML] Nanoparticle biodistribution coefficients: A quantitative approach for understanding the tissue distribution of nanoparticles

M Kumar, P Kulkarni, S Liu, N Chemuturi… - Advanced Drug Delivery …, 2023 - Elsevier
The objective of this manuscript is to provide quantitative insights into the tissue distribution
of nanoparticles. Published pharmacokinetics of nanoparticles in plasma, tumor and 13 …

Biocompatible iron oxide nanoparticles for targeted cancer gene therapy: A review

J Zhang, T Zhang, J Gao - Nanomaterials, 2022 - mdpi.com
In recent years, gene therapy has made remarkable achievements in tumor treatment. In a
successfully cancer gene therapy, a smart gene delivery system is necessary for both …

Autologous mesenchymal stem cells offer a new paradigm for salivary gland regeneration

M Marinkovic, ON Tran, H Wang… - International Journal of …, 2023 - nature.com
Salivary gland (SG) dysfunction, due to radiotherapy, disease, or aging, is a clinical
manifestation that has the potential to cause severe oral and/or systemic diseases and …

The perspective of DMPK on recombinant adeno-associated virus-based gene therapy: past learning, current support, and future contribution

N Chen, K Sun, NV Chemuturi, H Cho, CQ Xia - The AAPS Journal, 2022 - Springer
Given the recent success of gene therapy modalities and the growing number of cell and
gene-based therapies in clinical development across many different therapeutic areas, it is …

CPEB3 suppresses gastric cancer progression by inhibiting ADAR1-mediated RNA editing via localizing ADAR1 mRNA to P bodies

J Chen, L Li, TY Liu, HF Fu, YH Lai, X Lei, JF Xu, JS Yu… - Oncogene, 2022 - nature.com
Deciphering the crosstalk between RNA-binding proteins and corresponding RNAs will
provide a better understanding of gastric cancer (GC) progression. The comprehensive …

Allometric-like scaling of AAV gene therapy for systemic protein delivery

A Burr, P Erickson, R Bento, K Shama, C Roth… - … Therapy-Methods & …, 2022 - cell.com
The use of adeno-associated virus (AAV) as a gene delivery vehicle for secreted peptide
therapeutics can enable a new approach to durably manage chronic protein insufficiencies …

Structural characterization of porcine adeno-associated virus capsid protein with nuclear trafficking protein importin alpha reveals a bipartite nuclear localization signal

M Hoad, EM Cross, CM Donnelly, S Sarker, JA Roby… - Viruses, 2023 - mdpi.com
Adeno-associated viruses (AAV) are important vectors for gene therapy, and accordingly,
many aspects of their cell transduction pathway have been well characterized. However, the …

Prospects for gene replacement therapies in amyotrophic lateral sclerosis

I Giovannelli, A Higginbottom, J Kirby… - Nature Reviews …, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating and incurable neurodegenerative
disease characterized by the progressive loss of upper and lower motor neurons. ALS …

Modeling pharmacokinetics and pharmacodynamics of therapeutic antibodies: progress, challenges, and future directions

Y Tang, Y Cao - Pharmaceutics, 2021 - mdpi.com
With more than 90 approved drugs by 2020, therapeutic antibodies have played a central
role in shifting the treatment landscape of many diseases, including autoimmune disorders …