Evolution of nanomedicine formulations for targeted delivery and controlled release

S Puri, M Mazza, G Roy, RM England, L Zhou… - Advanced Drug Delivery …, 2023 - Elsevier
Nanotechnology research over the past several decades has been aimed primarily at
improving the physicochemical properties of small molecules to produce druggable …

Liver directed adeno‐associated viral vectors to treat metabolic disease

MA Chuecos, WR Lagor - Journal of Inherited Metabolic …, 2024 - Wiley Online Library
The liver is the metabolic center of the body and an ideal target for gene therapy of inherited
metabolic disorders (IMDs). Adeno‐associated viral (AAV) vectors can deliver transgenes to …

The recruitment of TRiC chaperonin in rotavirus viroplasms correlates with virus replication

J Vetter, G Papa, K Tobler, JM Rodriguez, M Kley… - Mbio, 2024 - Am Soc Microbiol
Rotavirus (RV) replication takes place in the viroplasms, cytosolic inclusions that allow the
synthesis of virus genome segments and their encapsidation in the core shell, followed by …

Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights

E Lopez-Gordo, K Chamberlain, JM Riyad… - Viruses, 2024 - mdpi.com
Today, adeno-associated virus (AAV)-based vectors are arguably the most promising in vivo
gene delivery vehicles for durable therapeutic gene expression. Advances in molecular …

Biophysical characterization of adeno-associated virus capsid through the viral transduction life cycle

YS Xiang, GG Hao - Journal of Genetic Engineering and Biotechnology, 2023 - Elsevier
Adeno-associated virus (AAV) vectors have emerged as the leading delivery platforms for
gene therapy. Throughout the life cycle of the virions, the capsid vector carries out diverse …

Interferon-γ inducible factor 16 (IFI16) restricts adeno-associated virus type 2 (AAV2) transduction in an immune-modulatory independent way

SO Sutter, K Tobler, M Seyffert, A Lkharrazi… - Journal of …, 2024 - Am Soc Microbiol
We determined the transcription profile of adeno-associated virus type 2 (AAV2)-infected
primary human fibroblasts. Subsequent analysis revealed that cells respond to AAV infection …

Fluorescence Microscopy in Adeno-Associated Virus Research

SK Golm, W Hübner, KM Müller - Viruses, 2023 - mdpi.com
Research on adeno-associated virus (AAV) and its recombinant vectors as well as on
fluorescence microscopy imaging is rapidly progressing driven by clinical applications and …

Adeno-Associated Virus Vectors—a Target of Cellular and Humoral Immunity—are Expanding Their Reach Toward Hematopoietic Stem Cell Modification and …

AE Araujo, M Bentler, X Perez Garmendia… - Human Gene …, 2024 - liebertpub.com
All current market-approved gene therapy medical products for in vivo gene therapy of
monogenic diseases rely on adeno-associated virus (AAV) vectors. Advances in gene …

Polyamine Dysregulation and Nucleolar Disruption in Alzheimer's Disease

WH Brooks - Journal of Alzheimer's Disease, 2024 - content.iospress.com
A hypothesis of Alzheimer's disease etiology is proposed describing how cellular stress
induces excessive polyamine synthesis and recycling which can disrupt nucleoli …

Multi-step engineered adeno-associated virus enables whole-brain mRNA delivery

W Bai, D Yang, Y Zhao, G Li, Z Liu, P Xiong, H Quan… - bioRxiv, 2024 - biorxiv.org
Adeno-associated viruses (AAVs) are commonly used vectors for DNA delivery in gene
therapy. Here we developed a system that enables the AAV shell to package mRNAs by …