Search Results

Development of ß-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000 births annually worldwide. SCD is an autosomal recessive disease resulting from a single point mutation in codon six of the ß-globin gene (HBB). Ex vivo ß-globin gene...

Authors: Annalisa Lattanzi , Joab Camarena , Premanjali Lahiri , Helen Segal , Waracharee Srifa , Christopher...

Single-cell metabolic profiling reveals subgroups of primary human hepatocytes showing heterogeneous responses to drug challenge

Xenobiotics are primarily metabolized by hepatocytes in the liver, and primary human hepatocytes (PHHs) are the gold standard model for the assessment of drug efficacy, safety and toxicity in the early phases of drug development. Recent advances in...

Authors: E. Sanchez-Quant, M. L. Richter, M. Colomé-Tatché , C.P. Martinez-Jimenez

Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression

Background Chimeric antigen receptor (CAR) T cells have demonstrated high clinical response rates against hematological malignancies (e.g., CD19+ cancers) buthave shown limited activity in patients with solid tumors. Recent work showed that precise...

Authors: Mueller KP, Piscopo NJ, Forsberg MH, Saraspe LA, Das A, Russell B, Smerchansky M, Cappabianca D, Shi...

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

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

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

Standard operating protocol of hepatic organoid differentiation from human induced pluripotent stem cells

Mature liver organoids are promising cell sources for research to understand the pathology underlying a variety of conditions affecting the liver, including end-stage chronic liver disease. Although several methods exist for the differentiation of...

Authors: Min Jung Kim, Jaeseo Lee , Seon Ju Mun, Myung Jin Son, Jung-Hyun Kim

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...

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...

Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

Sickle cell disease (SCD) is a monogenic disorder that affects millions worldwide. Allogeneic hematopoietic stem cell transplantation is the only available cure. Here, we demonstrate the use of CRISPR/Cas9 and a short single-stranded oligonucleotide...

Authors: So Hyun Park , Ciaran M Lee , Daniel P Dever , Timothy H Davis , Joab Camarena , Waracharee Srifa ,...

SST Peer Reviewed

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Development of ß-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Single-cell metabolic profiling reveals subgroups of primary human hepatocytes showing heterogeneous responses to drug challenge

Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression

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

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

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

Standard operating protocol of hepatic organoid differentiation from human induced pluripotent stem cells

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

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

Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease