Chemoenzymatic semisynthesis of proteins

RE Thompson, TW Muir - Chemical reviews, 2019 - ACS Publications
Protein semisynthesis—defined herein as the assembly of a protein from a combination of
synthetic and recombinant fragments—is a burgeoning field of chemical biology that has …

Nature-inspired protein ligation and its applications

R Pihl, Q Zheng, Y David - Nature Reviews Chemistry, 2023 - nature.com
The ability to manipulate the chemical composition of proteins and peptides has been
central to the development of improved polypeptide-based therapeutics and has enabled …

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

JM Levy, WH Yeh, N Pendse, JR Davis… - Nature biomedical …, 2020 - nature.com
The success of base editors for the study and treatment of genetic diseases depends on the
ability to deliver them in vivo to the relevant cell types. Delivery via adeno-associated viruses …

A molecular assembly phase transition and kinetic proofreading modulate Ras activation by SOS

WYC Huang, S Alvarez, Y Kondo, YK Lee, JK Chung… - Science, 2019 - science.org
The guanine nucleotide exchange factor (GEF) Son of Sevenless (SOS) is a key Ras
activator that is autoinhibited in the cytosol and activates upon membrane recruitment …

Inteins: nature's gift to protein chemists

NH Shah, TW Muir - Chemical science, 2014 - pubs.rsc.org
Inteins are auto-processing domains found in organisms from all domains of life. These
proteins carry out a process known as protein splicing, which is a multi-step biochemical …

Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin

H Tang, H Wang, S Wang, SW Hu, J Lv, M Xun, K Gao… - Human Genetics, 2023 - Springer
Mutations to the OTOF gene are among the most common reasons for auditory neuropathy.
Although cochlear implants are often effective in restoring sound transduction, there are …

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

P Tornabene, I Trapani, R Minopoli… - Science translational …, 2019 - science.org
Retinal gene therapy with adeno-associated viral (AAV) vectors holds promises for treating
inherited and noninherited diseases of the eye. Although clinical data suggest that retinal …

Design of a split intein with exceptional protein splicing activity

AJ Stevens, ZZ Brown, NH Shah, G Sekar… - Journal of the …, 2016 - ACS Publications
Protein trans-splicing (PTS) by split inteins has found widespread use in chemical biology
and biotechnology. Herein, we describe the use of a consensus design approach to …

A promiscuous split intein with expanded protein engineering applications

AJ Stevens, G Sekar, NH Shah… - Proceedings of the …, 2017 - National Acad Sciences
The protein trans-splicing (PTS) activity of naturally split inteins has found widespread use in
chemical biology and biotechnology. However, currently used naturally split inteins suffer …

Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss

O Shubina-Oleinik, C Nist-Lund, C French… - Science …, 2021 - science.org
Hearing loss affects an estimated 466 million people worldwide, with a substantial fraction
due to genetic causes. Approximately 16% of genetic hearing loss is caused by pathogenic …