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Orthotopic T-cell receptor replacement in primary human T cells using CRISPR-Cas9-mediatedhomology-directed repair

Adoptive T cell therapy using T-cell receptor (TCR)-engineered T cells allows to redirect T cell specificity and to target any antigen of interest. Here, we apply advanced genetic engineering using clustered regularly interspaced short palindromic...

Authors: Carolin Moosmann,* Thomas R. Mueller, Dirk H. Busch, and Kilian Schober

CRISPR-Cas9 cytidine and adenosine base editing of splice-sites mediates highly-efficient disruption of proteins in primary and immortalized cells

CRISPR-Cas9 cytidine and adenosine base editors (CBEs and ABEs) can disrupt genes without introducing double-stranded breaks by inactivating splice sites (BE-splice) or by introducing premature stop (pmSTOP) codons. However, no in-depth comparison of...

Authors: Kluesner MG, Lahr WS, Lonetree CL, Smeester BA, Qiu X, Slipek NJ, Claudio Vázquez PN, Pitzen SP,...

SST Peer Reviewed

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

Targeted T cell receptor gene editing provides predictable T cell product function for immunotherapy

Adoptive transfer of T cells expressing a transgenic T cell receptor (TCR) has the potential to revolutionize immunotherapy of infectious diseases and cancer. However, the generation of defined TCR-transgenic T cell medicinal products with...

Authors: Thomas R Müller , Sebastian Jarosch 1, Monika Hammel , Justin Leube , Simon Grassmann , Bettina...

SST Peer Reviewed

mRNA-mediated delivery of gene editing tools to human primary muscle stem cells

Muscular dystrophies are approximately 50 devastating, untreatable monogenic diseases leading to progressive muscle degeneration and atrophy. Gene correction of transplantable cells using CRISPR/Cas9-based tools is a realistic scenario for autologous...

Authors: Stadelmann C, Di Francescantonio S, Marg A, Müthel S, Spuler S, Escobar H

SST Peer Reviewed

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

Modeling retinitis pigmentosa through patient-derived retinal organoids

Human-induced pluripotent stem cells (hiPSCs) can be differentiated into well-structured retinal organoids. In this protocol, we successfully established 3D retinae from patient-derived hiPSCs and built the retinitis pigmentosa model in vitro....

Authors: Yan-Ping Li , Wen-Li Deng , Zi-Bing Jin

SST Peer Reviewed

High-efficiency nonviral CRISPR/Cas9-mediated gene editing of human T cells using plasmid donor DNA

Genome engineering of T lymphocytes, the main effectors of antitumor adaptive immune responses, has the potential to uncover unique insights into their functions and enable the development of next-generation adoptive T cell therapies. Viral gene...

Authors: Oh SA, Senger K, Madireddi S, Akhmetzyanova I, Ishizuka IE, Tarighat S, Lo JH, Shaw D, Haley B, Rutz...

SST Peer Reviewed

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

A functional genetic toolbox for human tissue-derived organoids

Human organoid systems recapitulate key features of organs offering platforms for modelling developmental biology and disease. Tissue-derived organoids have been widely used to study the impact of extrinsic niche factors on stem cells. However, they...

Authors: Dawei Sun , Lewis Evans , Francesca Perrone , Vanesa Sokleva , Kyungtae Lim , Saba Rezakhani ,...

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Orthotopic T-cell receptor replacement in primary human T cells using CRISPR-Cas9-mediatedhomology-directed repair

CRISPR-Cas9 cytidine and adenosine base editing of splice-sites mediates highly-efficient disruption of proteins in primary and immortalized cells

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

Targeted T cell receptor gene editing provides predictable T cell product function for immunotherapy

mRNA-mediated delivery of gene editing tools to human primary muscle stem cells

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

Modeling retinitis pigmentosa through patient-derived retinal organoids

High-efficiency nonviral CRISPR/Cas9-mediated gene editing of human T cells using plasmid donor DNA

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

A functional genetic toolbox for human tissue-derived organoids