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6404 results sorted by

Preclinical Activity of Embryonic Annexin A2-Specific Chimeric Antigen Receptor T Cells Against Ovarian Cancer

Chimeric antigen receptors (CARs) have found clinical success in B cell malignancies, but a dearth of potential targets limits their wider clinical application, especially in solid tumours. Here, we describe the development of an anti-annexin A2 CAR,...
Authors: Leonard Leong , Heng Liang Tan , Simeon Cua , Kylie Su Mei Yong , Qingfeng Chen , Andre Choo 
SST Peer Reviewed

TGF-ß inhibition via CRISPR promotes the long-term efficacy of CAR T cells against solid tumors

In recent years, chimeric antigen receptor-modified T cell (CAR T cell) therapy has proven to be a promising approach against cancer. Nonetheless, this approach still faces multiple challenges in eliminating solid tumors, one of which being the...
Authors: Tang N, Cheng C, Zhang X, Qiao M, Li N, Mu W, Wei XF, Han W, Wang H.

Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and their associated CRISPR-associated nucleases (Cas) are among the most promising technologies for the treatment of hemoglobinopathies including Sickle Cell Disease (SCD). We are...
Authors: Shirin R Modarai , Sambee Kanda , Kevin Bloh , Lynn M Opdenaker , Eric B Kmiec 
SST Peer Reviewed

Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing

A general approach for heritably altering gene expression has the potential to enable many discovery and therapeutic efforts. Here, we present CRISPRoff-a programmable epigenetic memory writer consisting of a single dead Cas9 fusion protein that...
Authors: Nuñez JK, Chen J, Pommier GC, Cogan JZ, Replogle JM, Adriaens C, Ramadoss GN, Shi Q, Hung KL,...

Nuclear Gene Transformation in the Dinoflagellate Oxyrrhis marina

The lack of a robust gene transformation tool that allows proper expression of foreign genes and functional testing for the vast number of nuclear genes in dinoflagellates has greatly hampered our understanding of the fundamental biology in this...
Authors: Brittany N Sprecher , Huan Zhang , Senjie Lin 

A CRISPR and high-content imaging assay compliant with ACMG/AMP guidelines for clinical variant interpretation in ciliopathies

Ciliopathies are a broad range of inherited developmental and degenerative diseases associated with structural or functional defects in motile or primary non-motile cilia. There are around 200 known ciliopathy disease genes and whilst genetic testing...
Authors: Nazlamova L, Thomas NS, Cheung MK, Legebeke J, Lord J, Pengelly RJ, Tapper WJ, Wheway G.
SST Peer Reviewed

Global post-translational modification profiling of HIV-1-infected cells reveals mechanisms of host cellular pathway remodeling

Viruses must effectively remodel host cellular pathways to replicate and evade immune defenses, and they must do so with limited genomic coding capacity. Targeting post-translational modification (PTM) pathways provides a mechanism by which viruses...
Authors: Jeffrey R. Johnson, David C. Crosby, Judd F. Hultquist, Donna Li, John Marlett, Justine Swann, Ruth...

CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

The prototypical genetic autoimmune disease is immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, a severe pediatric disease with limited treatment options. IPEX syndrome is caused by mutations in the forkhead box protein 3...
Authors: Goodwin M, Lee E, Lakshmanan U, Shipp S, Froessl L, Barzaghi F, Passerini L, Narula M, Sheikali A,...

Base-edited CAR T Cells for combinational therapy against T cell malignancies

Targeting T cell malignancies using chimeric antigen receptor (CAR) T cells is hindered by ‘T v T’ fratricide against shared antigens such as CD3 and CD7. Genome-editing can overcome such hurdles through targeted disruption of problematic shared...
Authors: Christos Georgiadis, Jane Rasaiyaah,  Soragia Athina Gkazi, Roland Preece, Aniekan Etuk, Abraham...

NUDT21 limits CD19 levels through alternativemRNA polyadenylation in B cell acutelymphoblastic leukemia

B cell progenitor acute lymphoblastic leukemia (B-ALL) treatment has been revolutionized by T cell-based immunotherapies-including chimeric antigen receptor T cell therapy (CAR-T) and the bispecific T cell engager therapeutic, blinatumomab-targeting...
Authors: Witkowski MT, Lee S, Wang E, Lee AK, Talbot A, Ma C, Tsopoulidis N, Brumbaugh J, Zhao Y, Roberts KG,...
SST Peer Reviewed
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