Induced pluripotent stem cells and their use in human models of disease and development

P Karagiannis, K Takahashi, M Saito… - Physiological …, 2019 - journals.physiology.org
The discovery of somatic cell nuclear transfer proved that somatic cells can carry the same
genetic code as the zygote, and that activating parts of this code are sufficient to reprogram …

[HTML][HTML] Current progress in therapeutic gene editing for monogenic diseases

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 for SCID-X1 in long-term hematopoietic stem cells

M Pavel-Dinu, V Wiebking, BT Dejene, W Srifa… - Nature …, 2019 - nature.com
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 …

Progenitor T-cell differentiation from hematopoietic stem cells using Delta-like-4 and VCAM-1

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 …

[HTML][HTML] Modeling human severe combined immunodeficiency and correction by CRISPR/Cas9-enhanced gene targeting

CW Chang, YS Lai, E Westin, A Khodadadi-Jamayran… - Cell reports, 2015 - cell.com
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 …

Stem cells and lung regeneration

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 …

Differentiation of human induced pluripotent stem cells into erythroid cells

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 …

Natural Killer Cells Generated From Human Induced Pluripotent Stem Cells Mature to CD56brightCD16+NKp80+/- In-Vitro and Express KIR2DL2/DL3 and …

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 …

Hematopoietic cells from pluripotent stem cells: hope and promise for the treatment of inherited blood disorders

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 …

[HTML][HTML] CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells

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 …