Genetics

One Shot of Gene Therapy and Children with Congenital Blindness Can Now See

gene therapy researchers

Source insciences.org Image: Albert M. Maguire, M.D; Katherine High, M.D.; and Jean Bennett, M.D., Ph.D., are co-authors of the study published in The Lancet. The gene therapy vector (shown) used in the study was manufactured at the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia. (Image from insciences.org)

PHILADELPHIA – Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.


molecules dna wrapping replication protein histone nucleus nuclei zone chromatin chromosome cell double helix

This animation shows a DNA wrapping, as well as it's replication.It was made by Drew Barry at The Walter & Eliza Hall Institute of Medical Research.


Surpassing the Law of Averages - How to expose the behaviors of genes, RNA, proteins, and metabolites in single cells.

Target With Dots

Source the-scientist.com Image from the-scientist.com

By necessity or convenience, almost everything we know about biochemistry and molecular biology derives from bulk behavior: From gene regulation to Michaelis-Menten kinetics, we understand biology in terms of what the “average” cell in a population does.

But, as Jonathan Weissman of the University of California, San Francisco, points out, “A lot gets lost in the average.” For instance, “The census might say, the average family has 1.9 children, but no family has 1.9 children.” Similarly, though most bacterial cells in a culture may behave in one way—growing preferentially on glucose, for instance—a small number may exist in a different state, the better to protect the population against the vagaries of future environmental conditions.


Synthetic DNA on the Brink of Yielding New Life Forms

DNA

It has been 50 years since scientists first created DNA in a test tube, stitching ordinary chemical ingredients together to make life's most extraordinary molecule. Until recently, however, even the most sophisticated laboratories could make only small snippets of DNA -- an extra gene or two to be inserted into corn plants, for example, to help the plants ward off insects or tolerate drought.

Now researchers are poised to cross a dramatic barrier: the creation of life forms driven by completely artificial DNA.

Scientists in Maryland have already built the world's first entirely handcrafted chromosome -- a large looping strand of DNA made from scratch in a laboratory, containing all the instructions a microbe needs to live and reproduce.


Genetic Risk Factor for Multiple Sclerosis

Multiple Sclerosis

A variant in a gene encoding a key regulator of the immune system increases the risk of multiple sclerosis, report two papers to be published online this week in Nature Genetics. Multiple sclerosis is a chronic and debilitating autoimmune disease in which neurons of the central nervous system become demyelinated, resulting in progressive neurodegeneration.


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