Two clinical trials using CRISPR technology to engineer the T cells of cancer patients by inactivating the gene for immune checkpoint molecule PD-1 are (almost) underway, one in the USA and the other in China. The less ...

The first therapy based on chimeric antigen receptors has been approved by the FDA. It gives excellent results in recurrent paediatric ALL; its cost is very high but fairly consistent with the clinical benefit (long term ...

Since the completion of the first human DNA sequence, genomic approaches have penetrated into cancer research and therapy: first through expression profiling for diagnostic, prognostic and predictive purposes, then by ...

The concept of precision oncology is straightforward, but the actual results of treating advanced cancer patients with the targeted drug suggested by genomic analysis have been generally disappointing, indicating that this ...

Recent efforts at gene therapy for haemophilia A and B using AAV-derived vectors show durable expression of coagulation factors at significant levels, resulting in almost complete correction of the phenotype. This is the ...

A recent paper shows that cancer patients using complementary therapies in addition to conventional treatment have distinctly worse outcomes. Analysis of the data shows that they are much more likely to refuse one component ...

Inflated drug prices necessarily raise the issue of rational allocation of health care resources. The system operated by the NICE agency in the UK attempts to do this by calculating the cost per quality-adjusted life year ...

Hutchinson-Gilford Progeria (acute premature aging) is caused by a de novo point mutation in the lamin A gene. Recently, this mutation has been accurately corrected by base editing in patient cell lines and in a mouse ...

In vivo inactivation of a deleterious gene has been achieved in a small trial, with excellent clinical results. Interestingly, the delivery and editing system is the same as in previous work on a different disease, and the ...