V Prakash, M Moore, RJ Yáñez-Muñoz - Molecular Therapy, 2016 - cell.com
Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome …
Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here we …
S Shukla, MA Langley, J Singh, JM Edgar… - Nature …, 2017 - nature.com
The molecular and cellular signals that guide T-cell development from hematopoietic stem and progenitor cells (HSPCs) remain poorly understood. The thymic microenvironment …
Mutations of the Janus family kinase JAK3 gene cause severe combined immunodeficiency (SCID). JAK3 deficiency in humans is characterized by the absence of circulating T cells and …
KR Parekh, J Nawroth, A Pai… - … of Physiology-Cell …, 2020 - journals.physiology.org
The ability to replace defective cells in an airway with cells that can engraft, integrate, and restore a functional epithelium could potentially cure a number of lung diseases. Progress …
M Ebrahimi, M Forouzesh, S Raoufi, M Ramazii… - Stem Cell Research & …, 2020 - Springer
During the last years, several strategies have been made to obtain mature erythrocytes or red blood cells (RBC) from the bone marrow or umbilical cord blood (UCB). However, UCB …
J Euchner, J Sprissler, T Cathomen, D Fürst… - Frontiers in …, 2021 - frontiersin.org
The differentiation of human induced pluripotent stem cells (hiPSCs) into T and natural killer (NK) lymphocytes opens novel possibilities for developmental studies of immune cells and …
I Rao, L Crisafulli, M Paulis, F Ficara - Cells, 2022 - mdpi.com
Inherited blood disorders comprise a large spectrum of diseases due to germline mutations in genes with key function in the hematopoietic system; they include immunodeficiencies …
EJ Kim, KH Kang, JH Ju - The Korean Journal of Internal Medicine, 2017 - ncbi.nlm.nih.gov
Recent advances in genome editing with programmable nucleases have opened up new avenues for multiple applications, from basic research to clinical therapy. The ease of use of …