[HTML][HTML] CRISPR-Cas systems: Overview, innovations and applications in human disease research and gene therapy

Y Xu, Z Li - Computational and structural biotechnology journal, 2020 - Elsevier
Genome editing is the modification of genomic DNA at a specific target site in a wide variety
of cell types and organisms, including insertion, deletion and replacement of DNA, resulting …

[HTML][HTML] Optimizing CRISPR/Cas9 precision: Mitigating off-target effects for safe integration with photodynamic and stem cell therapies in cancer treatment

JPJ Merlin, H Abrahamse - Biomedicine & Pharmacotherapy, 2024 - Elsevier
CRISPR/Cas9 precision genome editing has revolutionized cancer treatment by introducing
specific alterations to the cancer genome. But the therapeutic potential of CRISPR/Cas9 is …

Efficient, footprint-free human iPSC genome editing by consolidation of Cas9/CRISPR and piggyBac technologies

G Wang, L Yang, D Grishin, X Rios, LY Ye, Y Hu, K Li… - Nature protocols, 2017 - nature.com
Genome editing of human induced pluripotent stem cells (hiPSCs) offers unprecedented
opportunities for in vitro disease modeling and personalized cell replacement therapy. The …

Using iPS cells toward the understanding of Parkinson's disease

R Torrent, F De Angelis Rigotti, P Dell'Era… - Journal of clinical …, 2015 - mdpi.com
Cellular reprogramming of somatic cells to human pluripotent stem cells (iPSC) represents
an efficient tool for in vitro modeling of human brain diseases and provides an innovative …

Isogenic pairs of induced-pluripotent stem-derived endothelial cells identify DYRK1A/PPARG/EGR1 pathway is responsible for Down syndrome-associated …

H Suginobe, H Ishida, Y Ishii, K Ueda… - Human Molecular …, 2024 - academic.oup.com
Down syndrome (DS) is the most prevalent chromosomal disorder associated with a higher
incidence of pulmonary arterial hypertension (PAH). The dysfunction of vascular endothelial …

Gene editing and clonal isolation of human induced pluripotent stem cells using CRISPR/Cas9

S Yumlu, J Stumm, S Bashir, AK Dreyer, P Lisowski… - Methods, 2017 - Elsevier
Human induced pluripotent stem cells (hiPSCs) represent an ideal in vitro platform to study
human genetics and biology. The recent advent of programmable nucleases makes also the …

May I cut in? Gene editing approaches in human induced pluripotent stem cells

N Brookhouser, S Raman, C Potts, DA Brafman - Cells, 2017 - mdpi.com
In the decade since Yamanaka and colleagues described methods to reprogram somatic
cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have …

Animal use for science in Europe

M Daneshian, F Busquet, T Hartung, M Leist - 2015 - kops.uni-konstanz.de
To investigate long-term trends of animal use, the EU animal use statistics from the 15
countries that have been in the EU since 1995 plus respective data from Switzerland were …

Reprogramming patient-derived cells to study the epilepsies

JM Parent, SA Anderson - Nature neuroscience, 2015 - nature.com
The epilepsies and related disorders of brain circuitry present significant challenges
associated with the use of human cells to study disease mechanisms and develop new …

Deciphering the pathogenic consequences of chromosomal aberrations in human genetic disease

WP Kloosterman, R Hochstenbach - Molecular cytogenetics, 2014 - Springer
Chromosomal aberrations include translocations, deletions, duplications, inversions,
aneuploidies and complex rearrangements. They underlie genetic disease in roughly 15 …