Rare diseases, in totality, affect a significant proportion of the population and represent an
unmet medical need facing the scientific community. However, the treatment of individuals …

With the technology of reprogramming somatic cells by introducing defined transcription
factors that enables the generation of “induced pluripotent stem cells (iPSCs)” with …

Functional genomics studies of the immune system require genetic manipulations that
involve both deletion of target genes and addition of elements to proteins of interest …

Human induced pluripotent stem cell (hiPSC) technologies are powerful tools for modeling
development and disease, drug screening, and regenerative medicine. Faithful gene …

The clustered regularly interspaced short palindromic repeats-associated protein 9 or
CRISPR/Cas9 system is one of the hottest topics discussed lately due to its robustness and …

While challenging, identifying individuals displaying resilience to Alzheimer's disease (AD)
and understanding the underlying mechanism holds great promise for the development of …

Urea cycle disorders are incurable enzymopathies that affect nitrogen metabolism and
typically lead to hyperammonemia. Arginase deficiency is caused by biallelic mutations in …

The study of human diseases requires the development of modelling systems that accurately
replicate both the normal physiology and the progression of the disease. However …

Since the first reported generation of induced pluripotent stem cells (hiPSCs) from human
somatic cells, the stem cell field has emerged as a promising avenue for both modeling and …

Human induced pluripotent stem cell (hiPSC) technologies are powerful tools for modeling
development and disease, drug screening, and regenerative medicine. Faithful gene …