A CRISPR Way To Fix Faulty Genes

A CRISPR Way To Fix Faulty Genes
by Joe Palca
June 26, 2014
http://www.npr.org/blogs/health/2014/06/26/325213397/a-crispr-way-to-fix-faulty-genes

“It’s really powerful, it’s a really exciting development,” says Craig Mello of the University of Massachusetts Medical School.
He won the Nobel Prize in 2006 for a different technique that also lets scientists modify how genes work.
But, Mello says, this new genetic tool – known as CRISPR for clustered regularly interspersed short palindromic repeats — is more powerful, “because now you can essentially change a genome at will to almost anything you want. The sky’s the limit.”

therapies for people with genetic blood diseases like sickle cell disease and thalassemia.

original paper:
CRISPR-Cas systems for editing, regulating and targeting genomes
Nature Biotechnology  32, 347–355 (2014)
Jeffry D Sander & J Keith Joung
http://www.nature.com/nbt/journal/v32/n4/full/nbt.2842.html

related:
Americans Win Nobel for Work in Genetic Therapy
October 02, 2006
http://www.npr.org/templates/story/story.php?storyId=6179998

http://www.nature.com/news/crispr-the-disruptor-1.17673

Arteriolar niches maintain HSC quiescence

Arteriolar niches maintain haematopoietic stem cell quiescence
Nature (2013) Published online 09 October 2013
http://www.nature.com/nature/journal/vaop/ncurrent/full/nature12612.html
Yuya Kunisaki, et al.

Cell cycle quiescence is a critical feature contributing to haematopoietic stem cell (HSC) maintenance.

Although various candidate stromal cells have been identified as potential HSC niches, the spatial localization of quiescent HSCs in the bone marrow remains unclear.

Here, using a novel approach that combines whole-mount confocal immunofluorescence imaging techniques and computational modelling to analyse significant three-dimensional associations in the mouse bone marrow among vascular structures, stromal cells and HSCs, we show that quiescent HSCs associate specifically with small arterioles that are preferentially found in endosteal bone marrow.

These arterioles are ensheathed exclusively by rare NG2 (also known as CSPG4)+ pericytes, distinct from sinusoid-associated leptin receptor (LEPR)+ cells.