Systemic AAV vectors for widespread and targeted gene delivery in rodents

RC Challis, S Ravindra Kumar, KY Chan, C Challis… - Nature protocols, 2019 - nature.com
We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and
noninvasive gene transfer to the central and peripheral nervous systems. However, a …

A user's guide to the inverted terminal repeats of adeno-associated virus

P Wilmott, L Lisowski, IE Alexander… - Human gene therapy …, 2019 - liebertpub.com
Ongoing development of recombinant vectors based on adeno-associated virus (rAAV) is
providing an increasingly powerful and widely used toolkit for gene transfer and genome …

Self-attenuating adenovirus enables production of recombinant adeno-associated virus for high manufacturing yield without contamination

W Su, MI Patrício, MR Duffy, JM Krakowiak… - Nature …, 2022 - nature.com
Recombinant adeno-associated virus (rAAV) shows great promise for gene therapy,
however scalability, yield and quality remain significant issues. Here we describe an rAAV …

Next-generation sequencing (NGS) platforms: an exciting era of genome sequence analysis

B Meera Krishna, MA Khan, ST Khan - Microbial Genomics in Sustainable …, 2019 - Springer
DNA referred to as blueprint of life codes for the diversity and function of all the living
organisms. Determining DNA sequences of the living organisms not only gives an overview …

A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency

L Wang, Y Yang, C Breton, P Bell, M Li, J Zhang… - Science …, 2020 - science.org
Ornithine transcarbamylase (OTC) deficiency is an X-linked urea cycle disorder associated
with high mortality. Although a promising treatment for late-onset OTC deficiency, adeno …

[图书][B] Microbial Genomics in Sustainable Agroecosystems: Volume 2

V Tripathi, P Kumar, P Tripathi, A Kishore, M Kamle - 2019 - Springer
The chapter “Shared microbiome in different ecosystems: a meta-omics perspective”
explains recent progress in the sequencing technologies and other omics approaches that …

Widespread and targeted gene expression by systemic AAV vectors: Production, purification, and administration

RC Challis, SR Kumar, KY Chan, C Challis, MJ Jang… - BioRXiv, 2018 - biorxiv.org
We recently developed novel AAV capsids for efficient and noninvasive gene transfer across
the central and peripheral nervous systems. In this protocol, we describe how to produce …

[PDF][PDF] Profiling AAV vector heterogeneity & contaminants using next-generation sequencing methods

NT Tran, PWL Tai - Cell and Gene Therapy Insights, 2024 - cdn.insights.bio
AAVs were originally discovered in 1965 as 'virus-like'particles [1, 2]. AAVs belong to a class
of small, non-enveloped, dependoparvoviruses that rely on co-infection with helper viruses …

Accurate and rapid sequence analysis of adeno-associated virus plasmids by Illumina next-generation sequencing

A Saveliev, J Liu, M Li, L Hirata, C Latshaw… - Human Gene …, 2018 - liebertpub.com
Sequence validation of plasmid DNA is a crucial quality control step that must occur prior to
adeno-associated virus (AAV) vector packaging through plasmid transfection. AAV cis …

The history of mutational pressure changes during the evolution of adeno-associated viruses: A message to gene therapy and DNA-vaccine vectors designers

VV Khrustalev, TA Khrustaleva, AN Stojarov… - Infection, Genetics and …, 2020 - Elsevier
The use of virus-associated vectors for gene therapy and vaccination have emerged as safe
and effective delivery system. Like all other genetic materials, these vehicles are also prone …