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 …

Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

AAV-based in vivo gene therapy for neurological disorders

Q Ling, JA Herstine, A Bradbury, SJ Gray - Nature Reviews Drug …, 2023 - nature.com
Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

The AAV vector toolkit: poised at the clinical crossroads

A Asokan, DV Schaffer, RJ Samulski - Molecular Therapy, 2012 - cell.com
The discovery of naturally occurring adeno-associated virus (AAV) isolates in different
animal species and the generation of engineered AAV strains using molecular genetics …

Chemical diversity in the sialic acids and related α-keto acids: an evolutionary perspective

T Angata, A Varki - Chemical reviews, 2002 - ACS Publications
The glycan chains of vertebrate glycoconjugates are composed mostly of five-and six-
carbon sugars. One striking exception is the family of sialic acids (Sia), which are R-keto …

Adeno-associated virus (AAV) cell entry: structural insights

NL Meyer, MS Chapman - Trends in microbiology, 2022 - cell.com
Adeno-associated virus (AAV) is the leading vector in emerging treatments of inherited
diseases. Higher transduction efficiencies and cellular specificity are required for broader …

Adeno-associated virus serotypes: vector toolkit for human gene therapy

Z Wu, A Asokan, RJ Samulski - Molecular therapy, 2006 - cell.com
Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of
gene therapy in the past decade. The exponential progress of AAV-based vectors has been …

Sialic acid receptors of viruses

M Matrosovich, G Herrler, HD Klenk - … chemistry and biology II: tools and …, 2015 - Springer
Sialic acid linked to glycoproteins and gangliosides is used by many viruses as a receptor
for cell entry. These viruses include important human and animal pathogens, such as …

Adeno-associated virus (AAV) serotypes have distinctive interactions with domains of the cellular AAV receptor

S Pillay, W Zou, F Cheng, AS Puschnik… - Journal of …, 2017 - Am Soc Microbiol
Adeno-associated virus (AAV) entry is determined by its interactions with specific surface
glycans and a proteinaceous receptor (s). Adeno-associated virus receptor (AAVR)(also …

Gene therapy tools for brain diseases

S Ingusci, G Verlengia, M Soukupova… - Frontiers in …, 2019 - frontiersin.org
Neurological disorders affecting the central nervous system (CNS) are still incompletely
understood. Many of these disorders lack a cure and are seeking more specific and effective …