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tag rare diseases culture disease medicine

DNA molecule.
Finding DNA Tags in AAV Stacks
Mariella Bodemeier Loayza Careaga, PhD | Mar 7, 2024 | 8 min read
Ten years ago, scientists put DNA barcodes in AAV vectors, creating an approach that simplified, expedited, and streamlined AAV screening. 
Island Disease
Jef Akst | Aug 1, 2012 | 3 min read
People living on islands in the Norwegian Sea suffer from an unusually high rate of certain genetic diseases and health issues, making the population ripe for research.
FDA Approves Oral Drug for Fabry Disease
Ashley Yeager | Aug 13, 2018 | 2 min read
The medicine increases the activity of a deficient enzyme in certain patients with the condition.
Decoding the Cellular Secrets of the Endometrium 
Deanna MacNeil, PhD | Jan 16, 2024 | 3 min read
Endometrial organoids and single cell analyses helped uncover the molecular mechanisms of a rare uterine condition.
Illustration of DNA double helix with purple background.
Unbalanced Allele Expression Associated with Mutation, Disease
Natalia Mesa, PhD | Feb 2, 2023 | 5 min read
A study identifies nearly 3,000 genes where one allele tends to be expressed more than the other, but the findings ignited controversy in the field. 
Defining Rare Disorders: A Profile of Judith Hall
Anna Azvolinsky | Sep 1, 2019 | 8 min read
By bringing genetics into clinical medicine, the University of British Columbia medical geneticist helped to identify the gene mutations responsible for many rare diseases.
bacteria and DNA molecules on a purple background.
Engineering the Microbiome: CRISPR Leads the Way
Mariella Bodemeier Loayza Careaga, PhD | Mar 15, 2024 | 10+ min read
Scientists have genetically modified isolated microbes for decades. Now, using CRISPR, they intend to target entire microbiomes.
Telomeres in Disease
Rodrigo Calado and Neal Young | May 1, 2012 | 10+ min read
Telomeres have been linked to numerous diseases over the years, but how exactly short telomeres cause diseases and how medicine can prevent telomere erosion are still up for debate.
Exploring the Past, Present, and Future of Brain Organoids 
Niki Spahich, PhD | Feb 18, 2024 | 5 min read
Paola Arlotta seeks to understand the complex symphony of brain development in vitro by using organoid models.
The Breakthrough Prize ?Trophy
2024 Breakthrough Prizes in Life Sciences
Danielle Gerhard, PhD | Sep 14, 2023 | 10 min read
This year’s Breakthrough Prizes honor advances in CAR T cancer therapies, cystic fibrosis, and Parkinson’s disease.

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