Polymeric delivery of therapeutic nucleic acids
R Kumar, CF Santa Chalarca, MR Bockman… - Chemical …, 2021 - ACS Publications
The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …
[HTML][HTML] Advances in CRISPR therapeutics
The clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …
DNA repair pathway choices in CRISPR-Cas9-mediated genome editing
Many clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-
associated protein 9 (Cas9)-based genome editing technologies take advantage of Cas …
associated protein 9 (Cas9)-based genome editing technologies take advantage of Cas …
A decade of FDA-approved drugs (2010–2019): trends and future directions
A total of 378 novel drugs and 27 biosimilars approved by the US Food and Drug
Administration (FDA) between 2010 and 2019 were evaluated according to approval …
Administration (FDA) between 2010 and 2019 were evaluated according to approval …
[HTML][HTML] DNA damage: from threat to treatment
A Carusillo, C Mussolino - Cells, 2020 - mdpi.com
DNA is the source of genetic information, and preserving its integrity is essential in order to
sustain life. The genome is continuously threatened by different types of DNA lesions, such …
sustain life. The genome is continuously threatened by different types of DNA lesions, such …
[HTML][HTML] TP53-dependent toxicity of CRISPR/Cas9 cuts is differential across genomic loci and can confound genetic screening
CRISPR/Cas9 gene editing can inactivate genes in a precise manner. This process involves
DNA double-strand breaks (DSB), which may incur a loss of cell fitness. We hypothesize that …
DNA double-strand breaks (DSB), which may incur a loss of cell fitness. We hypothesize that …
[HTML][HTML] Delivery technologies for T cell gene editing: Applications in cancer immunotherapy
While initial approaches to adoptive T cell therapy relied on the identification and expansion
of rare tumour-reactive T cells, genetic engineering has transformed cancer immunotherapy …
of rare tumour-reactive T cells, genetic engineering has transformed cancer immunotherapy …
A novel Cas9 fusion protein promotes targeted genome editing with reduced mutational burden in primary human cells
A Carusillo, S Haider, R Schäfer, M Rhiel… - Nucleic Acids …, 2023 - academic.oup.com
Precise genome editing requires the resolution of nuclease-induced DNA double strand
breaks (DSBs) via the homology-directed repair (HDR) pathway. In mammals, this is …
breaks (DSBs) via the homology-directed repair (HDR) pathway. In mammals, this is …
[HTML][HTML] Emerging strategies in TCR-engineered T cells
Immunotherapy of cancer has made tremendous progress in recent years, as demonstrated
by the remarkable clinical responses obtained from adoptive cell transfer (ACT) of patient …
by the remarkable clinical responses obtained from adoptive cell transfer (ACT) of patient …
[HTML][HTML] Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases
MJ Schmidt, A Gupta, C Bednarski… - Nature …, 2021 - nature.com
Abstract Streptococcus pyogenes (Spy) Cas9 has potential as a component of gene
therapeutics for incurable diseases. One of its limitations is its large size, which impedes its …
therapeutics for incurable diseases. One of its limitations is its large size, which impedes its …