[HTML][HTML] Bioengineered AAV capsids with combined high human liver transduction in vivo and unique humoral seroreactivity

…, K Pekrun, E Zhu, S Nygaard, B Li, J Xu, K Chu… - Molecular Therapy, 2018 - cell.com
Existing recombinant adeno-associated virus (rAAV) serotypes for delivering in vivo gene
therapy treatments for human liver diseases have not yielded combined high-level human …

[HTML][HTML] The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner

A Gonzalez-Sandoval, K Pekrun, S Tsuji… - Nature …, 2023 - nature.com
… rAAV vectors were produced using a triple transfection protocol with PEI 25 K. Briefly, the
night before transfection, 293 T cells were seeded onto 15 cm 2 dishes (7.5e6 cells each dish …

[HTML][HTML] Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors

K Pekrun, G De Alencastro, QJ Luo, J Liu, Y Kim… - JCI insight, 2019 - ncbi.nlm.nih.gov
While gene transfer using recombinant adeno-associated viral (rAAV) vectors has shown
success in some clinical trials, there remain many tissues that are not well transduced. …

Semi-synthetic DNA shuffling of aveC leads to improved industrial scale production of doramectin by Streptomyces avermitilis

…, S Conlon, R Fedechko, H McArthur, K Pekrun… - Metabolic …, 2005 - Elsevier
The avermectin analog doramectin (CHC-B1), sold commercially as Dectomax TM , is
biosynthesized by Streptomyces avermitilis. aveC, a gene encoding an unknown mechanistic …

Molecular breeding of viruses

NW Soong, L Nomura, K Pekrun, M Reed… - Nature …, 2000 - nature.com
Genetic recombination is a major force driving the evolution of many viruses. Recombination
between two copackaged retroviral genomes may occur at rates as high as 40% per …

Fludarabine increases nuclease-free AAV-and CRISPR/Cas9-mediated homologous recombination in mice

…, H Jang, G de Alencastro, AF Muro, K Pekrun… - Nature …, 2022 - nature.com
Homologous recombination (HR)-based gene therapy using adeno-associated viruses (AAV-HR)
without nucleases has several advantages over classic gene therapy, especially the …

[HTML][HTML] Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model

R Song, K Pekrun, TA Khan, F Zhang, SP Paşca… - … Therapy-Methods & …, 2022 - cell.com
A limitation for recombinant adeno-associated virus (rAAV)-mediated gene transfer into the
central nervous system (CNS) is the low penetration of vectors across the human blood-brain …

Chromosome conformation paints reveal the role of lamina association in genome organization and regulation

…, MEG Sauria, X Wong, MC Gaillard, P Tsang, K Pekrun… - BioRxiv, 2017 - biorxiv.org
Non-random, dynamic three-dimensional organization of the nucleus is important for
regulation of gene expression. Numerous studies using chromosome conformation capture …

[HTML][HTML] Bioengineered viral platform for intramuscular passive vaccine delivery to human skeletal muscle

…, K Pekrun, GW Charville, K Maguire-Nguyen… - … Therapy-Methods & …, 2018 - cell.com
Skeletal muscle is ideal for passive vaccine administration as it is easily accessible by
intramuscular injection. Recombinant adeno-associated virus (rAAV) vectors are in consideration …

The repressive genome compartment is established early in the cell cycle before forming the lamina associated domains

…, X Wong, E DeBoy, MC Gaillard, P Tsang, K Pekrun… - BioRxiv, 2018 - biorxiv.org
… linearly ramped from 10000 K to 5000 K. At the same time, the k 2 parameter for constraint
… was linearly ramped down from 5000 K to 10 K. Each simulation was repeated 10 times and …