Search Results

CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells.

Human neural stem cells (NSCs) offer therapeutic potential for neurodegenerative diseases, such as inherited monogenic nervous system disorders, and neural injuries. Gene editing in NSCs (GE-NSCs) could enhance their therapeutic potential. We show...

Authors: Dever DP, Scharenberg SG, Camarena J, Kildebeck EJ, Clark JT, Martin RM, Bak RO, Tang Y, Dohse M,...

Genome-wide CRISPR Screens in Primary Human T Cells Reveal Key Regulators of Immune Function.

Human T cells are central effectors of immunity and cancer immunotherapy. CRISPR-based functional studies in T cells could prioritize novel targets for drug development and improve the design of genetically reprogrammed cell-based therapies. However,...

Authors: Shifrut E, Carnevale J, Tobin V, Roth TL, Woo JM, Bui CT, Li PJ, Diolaiti ME, Ashworth A, Marson...

SST Peer Reviewed

Generation of integration-free iPS cell lines from three sickle cell disease patients from the state of Bahia, Brazil

Sickle cell disease (SCD) is one of the most prevalent and severe monogenetic disorders, affecting several million people around the world. Clinical manifestationsand complications of the disease include sickle cell pain crisis, silent cerebral...

Authors: Martins GLS, Paredes BD, Azevedo CM, Sampaio GLA, Nonaka CKV, Cavalcante BRR, Da Silva KN, Pereira...

SST Peer Reviewed

CRISPR-Cas9 genome engineering of primary CD4+ T cells for the interrogation of HIV-host factor interactions.

CRISPR-Cas9 gene-editing strategies have revolutionized our ability to engineer the human genome for robust functional interrogation of complex biological processes. We have recently adapted this technology for use in primary human CD4+ T cells to...

Authors: Hultquist JF, Hiatt J, Schumann K, McGregor MJ, Roth TL, Haas P, Doudna JA, Marson A, Krogan NJ.

SST Peer Reviewed

Induced Neurons for the Study of Neurodegenerative and Neurodevelopmental Disorders.

Patient-derived or genomically modified human induced pluripotent stem cells (iPSCs) offer the opportunity to study neurodevelopmental and neurodegenerative disorders. Overexpression of certain neurogenic transcription factors (TFs) in iPSCs can...

Authors: Sauter EJ, Kutsche LK, Klapper SD, Busskamp V.

SST Peer Reviewed

Generation of three control iPS cell lines for sickle cell disease studies by reprogramming erythroblasts from individuals without hemoglobinopathies

Sickle cell disease (SCD) is one of the most prevalent and severe monogenetic disorders. Previously, we generated iPS cell lines from SCD patients. Here, we generatediPS cell lines from three age-, ethnicity- and gender-matched healthy individuals as...

Authors: Paredes BD, Martins GLS, Azevedo CM, Sampaio GLA, Nonaka CKV, Silva KND, Soares MBP, Santos...

SST Peer Reviewed

Flow Cytometry Based Detection and Isolation of Plasmodium falciparum Liver Stages In Vitro

Malaria, the disease caused by Plasmodium parasites, remains a major global health burden. The liver stage of Plasmodium falciparuminfection is a leading target for immunological and pharmacological interventions....

Authors: Dumoulin PC, Trop SA, Ma J, Zhang H, Sherman MA, Levitskaya J

Human blood vessel organoids as a model of diabetic vasculopathy

The increasing prevalence of diabetes has resulted in a global epidemic1. Diabetes is a major cause of blindness, kidney failure, heart attacks, stroke and amputation of lower limbs. These are often caused by changes in blood vessels, such as the...

Authors: Wimmer RA, Leopoldi A, Aichinger M, Wick N, Hantusch B, Novatchkova M, Taubenschmid J, Hämmerle M,...

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Re-expression of the paralogous gamma-globin genes (HBG1/2) could be a universal strategy to ameliorate the severe ß-globin disorders sickle cell disease (SCD) and ß-thalassemia by induction of fetal hemoglobin (HbF, a2?2)1. Previously, we and others...

Authors: Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen...

SST Peer Reviewed

Development of glial restricted human neural stem cells for oligodendrocyte differentiation in vitro and in vivo.

In this study, we have developed highly expandable neural stem cells (NSCs) from HESCs and iPSCs that artificially express the oligodendrocyte (OL) specific transcription factor gene Zfp488. This is enough to restrict them to an exclusive...

Authors: Biswas S Chung SH Jiang P Dhaghan S Deng W

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CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells.

Genome-wide CRISPR Screens in Primary Human T Cells Reveal Key Regulators of Immune Function.

Generation of integration-free iPS cell lines from three sickle cell disease patients from the state of Bahia, Brazil

CRISPR-Cas9 genome engineering of primary CD4+ T cells for the interrogation of HIV-host factor interactions.

Induced Neurons for the Study of Neurodegenerative and Neurodevelopmental Disorders.

Generation of three control iPS cell lines for sickle cell disease studies by reprogramming erythroblasts from individuals without hemoglobinopathies

Flow Cytometry Based Detection and Isolation of Plasmodium falciparum Liver Stages In Vitro

Human blood vessel organoids as a model of diabetic vasculopathy

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Development of glial restricted human neural stem cells for oligodendrocyte differentiation in vitro and in vivo.