There are few better examples of the need for data sharing than in the rare disease community,
where patients, physicians, and researchers must search for “the needle in a haystack” to …

Targeted delivery of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated
protein 9 (Cas9) system to the receptor cells is essential for in vivo gene …

Defects in primary cilium biogenesis underlie the ciliopathies, a growing group of genetic
disorders. We describe a whole-genome siRNA-based reverse genetics screen for defects in …

Joubert syndrome related disorders (JSRDs) have broad but variable phenotypic overlap
with other ciliopathies. The molecular etiology of this overlap is unclear but probably arises …

Mandibulofacial dysostosis with microcephaly (MFDM) is a rare sporadic syndrome comprising
craniofacial malformations, microcephaly, developmental delay, and a recognizable …

Biomaterials as bone substitutes are always considered as foreign bodies that can trigger
host immune responses. Traditional designing principles have been always aimed at …

Background Congenital nonprogressive spinocerebellar ataxia is characterized by early
gross motor delay, hypotonia, gait ataxia, mild dysarthria and dysmetria. The clinical …

To improve their low-temperature desulfurization performance, the activated carbon-based
materials have typically been modified by either metal oxide loading or nitrogen doping. …

Enterococcus faecalis rank among the leading causes of nosocomial infections worldwide
and possesses both intrinsic and acquired resistance to a variety of antibiotics. Development …

Inflammatory macrophages play pivotal roles in the development of atherosclerosis.
Theranostics, a promising approach for local imaging and photothermal therapy of inflammatory …