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tag tay sachs disease disease medicine

Gene Therapy Targets Canavan Disease
Douglas Steinberg | Sep 16, 2001 | 6 min read
The Canavan trial signals a new phase in a 10-year offensive that gene therapy researchers have waged against neurodegenerative disorders.
A Genetic Checkup: Lessons from Huntington Disease and Cystic Fibrosis
Ricki Lewis | Oct 19, 2003 | 9 min read
Thom Graves Media While genome sequencing may be the new kid on the block--perhaps now with a cracking voice and fuzzy facial hair--predicting phenotypes is the stuff of classical genetics, honed on the rare single-gene disorders, such as Huntington disease (HD) and cystic fibrosis (CF), which dominated the field in the last century (see Genetic Testing Timeline). "Geneticists today are portrayed as soothsayers of the future. But predictive medicine and testing has a significant history," says
Panel Lays Out Guidelines for CRISPR-Edited Human Embryos
Lisa Winter | Sep 4, 2020 | 2 min read
The International Commission on the Clinical Use of Human Germline Genome Editing claims the technology is still too risky for therapeutic use.
prime editing crispr cas9 genome editing techniques
New “Prime Editing” Method Makes Only Single-Stranded DNA Cuts
Emma Yasinski | Oct 21, 2019 | 4 min read
In demonstrations in cell lines, the technique has a similar efficacy to CRISPR-Cas9, but fewer off-target effects.
Glycobiology Goes to the Ball
Jeffrey Perkel | Apr 28, 2002 | 8 min read
There's more to life than DNA, RNA, and proteins. Literally. Sugars are also in the mix. And the roles that carbohydrates play in biology are just as important as those of any member of the better-characterized trinity. These macromolecules affect cell-cell interactions, immune function, and protein regulation, and disruption of their biology results in disease. One magazine likened the study of carbohydrates, called glycobiology, to Cinderella—neglected stepsister to her two more glamoro
Race and the Clinic: Good Science?
Ricki Lewis | Feb 17, 2002 | 8 min read
Humans have long embraced the idea of grouping and naming people who have distinct, genetically determined physical characteristics, like almond-shaped eyes or different skin color. It made sense, from a social standpoint (think safety, politics, and business) to align one's self with kin. However, studying race from a biological point of view, in the hopes of learning about specific diseases or developing new drugs, is a different matter altogether. "Race is generally not a useful consideration
Genetics-Based Testing Could Create A Biologic Underclass
Dorothy Nelkin | Nov 26, 1989 | 9 min read
[Editor’s note: Sophisticated biological tests that can uncover latent problems or predict future diseases have been developed over the past few years. Such tests have important clinical applications, but they also have found their way into nonclinical contexts in which they provide unprecedented threats to our traditional concepts of privacy and personal autonomy. So say Dorothy Nelkin and Laurence Tancredi, coauthors of a new book, Dangerous Diagnostics: The Social Power of Biological I
Considering Gene Editing
Jef Akst | Jul 12, 2016 | 3 min read
An international committee continues its investigation into the ethical and social considerations of precision DNA editing technology with a public meeting held this morning in Washington, DC.
Famed Geneticist Dies
Jef Akst | May 31, 2012 | 1 min read
David Rimoin, a medical geneticist and the founding president of the American College of Medical Genetics and Genomics, passes away at age 75.
Discoverer of Lysosomes Dies
Kate Yandell | May 8, 2013 | 2 min read
Christian de Duve chose to be euthanized at home in Belgium at age 95.

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