Rare diseases affect 30 million people in the USA and more than 300–400 million
worldwide, often causing chronic illness, disability, and premature death. Traditional …

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 …

Experimental modelling of human disorders enables the definition of the cellular and
molecular mechanisms underlying diseases and the development of therapies for treating …

The discovery of induced pluripotent stem cells (iPSCs) has made an invaluable contribution
to the field of regenerative medicine, paving way for identifying the true potential of human …

Despite its complexity, blood is probably the best understood developmental system, largely
due to seminal experimentation in the mouse. Clinically, hematopoietic stem cell (HSC) …

To date, over 1800 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Interstrand crosslinks (ICLs) are highly toxic DNA lesions that prevent transcription and
replication by inhibiting DNA strand separation. Agents that induce ICLs were one of the …

The function of Fanconi anaemia proteins is to maintain genomic stability. Their main role is
in the repair of DNA interstrand crosslinks, which, by covalently binding the Watson and the …

Background Long-QT syndromes are heritable diseases associated with prolongation of the
QT interval on an electrocardiogram and a high risk of sudden cardiac death due to …

The prospect of changing the plasticity of terminally differentiated cells toward pluripotency
has completely altered the outlook for biomedical research. Human-induced pluripotent …