From Alzheimer’s disease and the obesity epidemic to the chronic health effects of certain viral infections, scientists in academia and big pharma are seeking new therapeutic targets and strategies to tackle these diseases. However, as discussed in this Molecular Medicine Select, designing more effective drugs with fewer side effects that fight the disease itself and not just the symptoms requi...

The recent generation of induced pluripotent stem (iPS) cells from adult human somatic cells has intensified interest in cellular therapies for treating human disease. This issue’s Molecular Medicine Select highlights progress in overcoming barriers to the use of transplanted cells. These achievements include the treatment of sickle cell anemia in mice using genetically repaired iPS cells, the ...

Vestibular schwannomas, also known as acoustic neuromas, are benign tumors, which originate from myelin‐forming Schwann cells. They develop in the vestibular branch of the eighth cranial nerve in the internal auditory canal or cerebellopontine angle. The clinical progression of the condition involves slow and progressive growth, eventu‐ ally resulting in brainstem compression. The objective of ...

The current study was performed with aim to investigate the fibroblast growth factor receptor 2 (FGFR2) gene in two Chinese families with two different forms of syndromic craniosynostosis, and to characterize their asso‐ ciated clinical features. Two families underwent complete ophthalmic examinations, and two patients from each family were diagnosed with craniosynostosis. Genomic DNA was extra...

Immune responses are subject to many layers of regulation such that their effect is precisely calibrated. New approaches to selectively alter the intensity and type of immune response for therapeutic benefit are the topic of this issue’s Molecular Medicine Select. This includes the recent discovery of a modifier gene that shapes the activity of lung neutrophils to affect the severity of cystic ...

Glaucoma is a worldwide disease and the second leading cause of blindness. Current treatments are associated with a number of side-effects and poor compliance, due to the requirement for treatment administration several times a day. These treatments typically aim to lower intraocular pressure (IOP); however, they are unable to protect retinal ganglion cells (RGCs) from undergoing apoptosis, whi...

Recently, genome-wide association studies have linked variations in alleles of the FTO (fat mass and obesity associated) gene to obesity. Gerken et al. (2007) now reveal that FTO encodes a nucleic acid demethylase. Upon examination of the sequence of FTO, the authors noticed a domain that had homology to 2-oxoglutarate oxygenases. These enzymes are involved in a variety of processes including D...

Much progress has been made in identifying the factors that cause tumor formation. Yet, how cancer cells leave primary tumors to colonize distant organs—a process called metastasis—is still poorly understood. Here we discuss recent advances that describe factors that promote the invasive ability of cancer cells. MicroRNAs (miRNAs) have emerged as key regulators of many biological processes, bot...