Results 21 to 30 of about 321,595 (358)

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

New England Journal of Medicine, 2021
BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart.
J. Gillmore, E. Gane, J. Taubel, J. Kao, M. Fontana, M. Maitland, Jessica Seitzer, D. O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, A. Boyd, J. Cehelsky, M. McKee, A. Schiermeier, O. Harari, A. Murphy, C. Kyratsous, B. Zambrowicz, Randy Soltys, D. Gutstein, J. Leonard, L. Sepp-Lorenzino, D. Lebwohl   +25 more
semanticscholar   +1 more source

Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9

Nature Biotechnology, 2015
CRISPR-Cas9–based genetic screens are a powerful new tool in biology. By simply altering the sequence of the single-guide RNA (sgRNA), one can reprogram Cas9 to target different sites in the genome with relative ease, but the on-target activity and off ...
John G Doench, Nicoló Fusi, Meagan E. Sullender, Mudra Hegde, Emma Vaimberg, Katherine F. Donovan, Ian Smith, Z. Tothova, Craig B. Wilen, R. Orchard, H. Virgin, J. Listgarten, D. Root   +12 more
semanticscholar   +1 more source

Today's Biothreats – Where the Past Predictions Meet the Future

Вестник войск РХБ защиты, 2020
The catastrophic spread of COVID-19 pandemic, uncontrolled by modern medical science, regardless of whether it is artificial or not, clearly shows the limits of human mind and knowledge to resist this and other similar challenges.
J. Lakota
doaj   +1 more source

CRISPR-engineered T cells in patients with refractory cancer

Science, 2020
CRISPR takes first steps in humans CRISPR-Cas9 is a revolutionary gene-editing technology that offers the potential to treat diseases such as cancer, but the effects of CRISPR in patients are currently unknown. Stadtmauer et al. report a phase 1 clinical
E. Stadtmauer, J. Fraietta, M. Davis, A. Cohen, Kristy Weber, E. Lancaster, P. Mangan, I. Kulikovskaya, Minnal Gupta, Fang Chen, Lifeng Tian, V. Gonzalez, Jun Xu, In-Young Jung, J. Melenhorst, G. Plesa, J. Shea, Tina Matlawski, A. Cervini, A. Gaymon, Stephanie Desjardins, Anne Lamontagne, January Salas-McKee, A. Fesnak, D. Siegel, B. Levine, J. Jadlowsky, R. Young, A. Chew, W. Hwang, E. Hexner, B. Carreno, Christopher L. Nobles, F. Bushman, Kevin R. Parker, Yanyan Qi, Ansuman T. Satpathy, Howard Y. Chang, Yangbing Zhao, S. Lacey, C. June   +40 more
semanticscholar   +1 more source

Desarrollo de nuevos diseños vegetales: Mejora genética vs transformación genética y edición génica

Magna Scientia UCEVA, 2021
El objetivo de esta reflexión es realizar un estudio comparativo sobre la eficacia, eficiencia técnica y costo de las tecnologías más importantes para el desarrollo de nuevos diseños vegetales, incluyendo la mejora genética, la transformación genética y ...
Pedro Martínez Gómez
doaj   +1 more source

Current trends and new methods of detection of SARS-CoV-2 infection [PDF]

Cellular, Molecular and Biomedical Reports, 2022
SARS-CoV- 2 is the causative agent of the global pandemic, also known as Covid-19. This virus belongs to a group of coronaviruses and has affected more than ten million people across the globe, causing nearly half a million deaths worldwide. The pandemic
Mohammad Mohammadi, Amir Omidi, Hoda Sabati   +2 more
doaj   +1 more source

High frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

Nature Biotechnology, 2013
Clustered, regularly interspaced, short palindromic repeat (CRISPR) RNA-guided nucleases (RGNs) have rapidly emerged as a facile and efficient platform for genome editing. Here, we use a human cell–based reporter assay to characterize off-target cleavage
Yanfang Fu, Jennifer A. Foden, Cyd Khayter, Morgan L. Maeder, D. Reyon, J., Keith Joung, Jeffry D. Sander   +7 more
semanticscholar   +1 more source

The influence of the copy number of invader on the fate of bacterial host cells in the antiviral defense by CRISPR-Cas10 DNases

Engineering Microbiology, 2023
Type III CRISPR-Cas10 systems employ multiple immune activities to defend their hosts against invasion from mobile genetic elements (MGEs), including DNase and cyclic oligoadenylates (cOA) synthesis both of which are hosted by the type-specific protein ...
Zhenxiao Yu, Jianan Xu, Yan Zhang, Qunxin She   +3 more
doaj   +1 more source

CHOPCHOP v3: expanding the CRISPR web toolbox beyond genome editing

Nucleic Acids Res., 2019
The CRISPR–Cas system is a powerful genome editing tool that functions in a diverse array of organisms and cell types. The technology was initially developed to induce targeted mutations in DNA, but CRISPR–Cas has now been adapted to target nucleic acids
Kornel Labun, Tessa G. Montague, M. Krause, Y. T. Cleuren, Håkon Tjeldnes, Eivind Valen   +5 more
semanticscholar   +1 more source

A human rights analysis of clustered regularly interspaced short palindromic repeats germline-editing for disease prevention

Journal of Public Health and Primary Care, 2020
Our purpose is to discuss ethical concerns with disease prevention applications of CRISPR germline editing using a human rights approach. If applied towards furthering health, these tools may aid to prevent diseases, thereby improving health and reduce ...
Lance Garrett Shaver, Amit Sundly, Abdullah Omar Saif   +2 more
doaj   +1 more source