Building cross-border collaborations to increase diversity and accelerate rare disease drug development - meeting report from the inaugural IndoUSrare Annual Conference 2021.

Unlabelled: The inaugural IndoUSrare Annual Conference was held virtually from 29 November to 2 December 2021 and was organized by the Indo US Organization for Rare Diseases (IndoUSrare). The event saw participation from over 250 stakeholders of rare diseases who joined in virtually by audio/video on the Zoom platform from around the world, with a majority of attendees concentrated in the Indian subcontinent and the United States. The conference was held over 4 days from 10:00 a.

A transcriptionally repressed quiescence program is associated with paused RNA polymerase II and is poised for cell cycle re-entry.

Adult stem cells persist in mammalian tissues by entering a state of reversible quiescence, referred to as G0, which is associated with low levels of transcription. Using cultured myoblasts and muscle stem cells, we report that in G0, global RNA content and synthesis are substantially repressed, correlating with decreased RNA polymerase II (RNAPII) expression and activation. Integrating RNAPII occupancy and transcriptome profiling, we identify repressed networks and a role for promoter-proximal RNAPII pausing in G0.

A comparative analysis of important public clinical trial registries, and a proposal for an interim ideal one.

Background: It is an ethical and scientific obligation to register each clinical trial, and report its results, accurately, comprehensively and on time. The WHO recognizes 17 public registries as Primary Registries, and has also introduced a set of minimal standards in the International Standards for Clinical Trial Registries (ISCTR) that primary registries need to implement. These standards are categorized into nine sections-Content, Quality and Validity, Accessibility, Unambiguous Identification, Technical Capacity, Administration and Governance, the Trial Registration Data Set (TRDS), Partner registries and Data Interchange Standards.

The primary cilium dampens proliferative signaling and represses a G2/M transcriptional network in quiescent myoblasts.

Background: Reversible cell cycle arrest (quiescence/G0) is characteristic of adult stem cells and is actively controlled at multiple levels. Quiescent cells also extend a primary cilium, which functions as a signaling hub. Primary cilia have been shown to be important in multiple developmental processes, and are implicated in numerous developmental disorders.

Mimicking Muscle Stem Cell Quiescence in Culture: Methods for Synchronization in Reversible Arrest.

Growing evidence supports the view that in adult stem cells, the defining stem cell features of potency and self-renewal are associated with the quiescent state. Thus, uncovering the molecular logic of this reversibly arrested state underlies not only a fundamental understanding of adult tissue dynamics but also hopes for therapeutic regeneration and rejuvenation of damaged or aging tissue. A key question concerns how adult stem cells use quiescence to establish or reinforce the property of self-renewal.