Search Results

Restriction of HIV-1-based lentiviral vectors in adult primary marrow-derived and peripheral mobilized human CD34+ hematopoietic stem and progenitor cells occurs prior to viral DNA integration

BACKGROUND: Gene therapy is currently being attempted using a number of delivery vehicles including lentiviral-based vectors. The delivery and insertion of a gene using lentiviral-based vectors involves multiple discrete steps, including reverse...

Authors: Griffin DO, Goff SP

SST Peer Reviewed

An optimized multiplex flow cytometry protocol for the analysis of intracellular signaling in peripheral blood mononuclear cells.

Phosphoflow cytometry is increasingly being used as a tool for the discovery of biomarkers used in the treatment and monitoring of disease and therapy. The ability to measure numerous phospho-protein targets simultaneously at a single cell level...

Authors: Davies R, Vogelsang P, Jonsson R, Appel S.

Genome Editing in Human Stem Cells

The use of custom-engineered sequence-specific nucleases (including CRISPR/Cas9, ZFN, and TALEN) allows genetic changes in human cells to be easily made with much greater efficiency and precision than before. Engineered double-stranded DNA breaks can...

Authors: Susan M. Byrne, Prashant Mali, and George M. Church

SST Peer Reviewed

Pelleted Bone Marrow Derived Mesenchymal Stem Cells Are Better Protected from the Deleterious Effects of Arthroscopic Heat Shock

INTRODUCTION: The impact of arthroscopic temperature on joint tissues is poorly understood and it is not known how mesenchymal stem cells (MSCs) respond to the effects of heat generated by the device during the process of arthroscopy assisted...

Authors: Kalamegam G, Abbas M, Gari M, Alsehli H, Kadam R, Alkaff M, Chaudhary A, Al-Qahtani M, Abuzenadah A,...

Loss of fibronectin from the aged stem cell niche affects the regenerative capacity of skeletal muscle in mice.

Age-related changes in the niche have long been postulated to impair the function of somatic stem cells. Here we demonstrate that the aged stem cell niche in skeletal muscle contains substantially reduced levels of fibronectin (FN), leading to...

Authors: Lukjanenko L, Jung MJ, Hegde N, Perruisseau-Carrier C, Migliavacca E, Rozo M, Karaz S, Jacot G,...

Insights into the role of sulfated glycans in cancer cell adhesion and migration through use of branched peptide probe

The tetra-branched peptide NT4 selectively binds to different human cancer cells and tissues. NT4 specifically binds to sulfated glycosaminoglycans on cancer cell membranes. Since sulfated glycosaminoglycans are involved in cancer cell interaction...

Authors: Brunetti J, Depau L, Falciani C, Gentile M, Mandarini E, Riolo G, Lupetti P, Pini A, Bracci L

SST Peer Reviewed

The new frontier of genome engineering with CRISPR-Cas9

The advent of facile genome engineering using the bacterial RNA-guided CRISPR-Cas9 system in animals and plants is transforming biology. We review the history of CRISPR (clustered regularly interspaced palindromic repeat) biology from its initial...

Authors: Jennifer A. Doudna, Emmanuelle Charpentier

SST Peer Reviewed

Manufacture of clinical-grade CD19-specific T cells stably expressing chimeric antigen receptor using Sleeping Beauty system and artificial antigen presenting cells.

Adoptive transfer of T cells expressing a CD19-specific chimeric antigen receptor (CAR) is being evaluated in multiple clinical trials. Our current approach to adoptive immunotherapy is based on a second generation CAR (designated CD19RCD28) that...

Authors: Singh H, Figliola MJ, Dawson MJ, Olivares S, Zhang L, Yang G, Maiti S, Manuri P, Senyukov V, Jena B,...

SST Peer Reviewed

High-efficiency genome editing via 2A-coupled co-expression of fluorescent proteins and zinc finger nucleases or CRISPR/Cas9 nickase pairs.

Targeted endonucleases including zinc finger nucleases (ZFNs) and clustered regularly interspaced short palindromic repeats (CRISPRs)/Cas9 are increasingly being used for genome editing in higher species. We therefore devised a broadly applicable and...

Authors: Duda K, Lonowski LA, Kofoed-Nielsen M, Ibarra A, Delay CM, Kang Q, Yang Z, Pruett-Miller SM, Bennett...

SST Peer Reviewed

Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.

Diseases of lipid metabolism are a major cause of human morbidity, but no animal model entirely recapitulates human lipoprotein metabolism. Here we develop a xenograft mouse model using hepatocytes from a patient with familial hypercholesterolaemia...

Authors: Bissig-Choisat B1, Wang L2, Legras X1, Saha PK3, Chen L1, Bell P2, Pankowicz FP4, Hill MC5, Barzi...

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Restriction of HIV-1-based lentiviral vectors in adult primary marrow-derived and peripheral mobilized human CD34+ hematopoietic stem and progenitor cells occurs prior to viral DNA integration

An optimized multiplex flow cytometry protocol for the analysis of intracellular signaling in peripheral blood mononuclear cells.

Genome Editing in Human Stem Cells

Pelleted Bone Marrow Derived Mesenchymal Stem Cells Are Better Protected from the Deleterious Effects of Arthroscopic Heat Shock

Loss of fibronectin from the aged stem cell niche affects the regenerative capacity of skeletal muscle in mice.

Insights into the role of sulfated glycans in cancer cell adhesion and migration through use of branched peptide probe

The new frontier of genome engineering with CRISPR-Cas9

Manufacture of clinical-grade CD19-specific T cells stably expressing chimeric antigen receptor using Sleeping Beauty system and artificial antigen presenting cells.

High-efficiency genome editing via 2A-coupled co-expression of fluorescent proteins and zinc finger nucleases or CRISPR/Cas9 nickase pairs.

Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.