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tag rare diseases disease medicine developmental biology ecology

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. 
Cropped view of senior man playing with puzzles
A Rare Genetic Mutation Protects Against Alzheimer's Disease
Hannah Thomasy, PhD, Drug Discovery News | Sep 17, 2023 | 4 min read
Data from a highly resilient individual guided researchers to new potential therapeutic targets.
The Challenges of Rare-Disease Research
Jyoti Madhusoodanan | Sep 1, 2016 | 8 min read
With few resources and hesitant investors, basic scientists must rely on clinicians, patient advocates, and their own keen eye for biological connections.
A three-dimensional rendered image of neuron cell network on black background.
Assembloids Unlock the Roles of Key Neurodevelopment Disease Genes
Aparna Nathan, PhD | Sep 27, 2023 | 3 min read
Brain-like tissue grown in a dish mimics critical periods for development and reveals how it can go wrong.
Valerie Arboleda Uses Big Data to Unravel the Biology of a Rare Disease
Shawna Williams | May 1, 2018 | 3 min read
The UCLA geneticist examines how defects in a histone protein lead to symptoms throughout the body.
Stem Cell Trial for Eye Disease Commences
Jef Akst | Sep 12, 2014 | 2 min read
Researchers at the RIKEN Center for Developmental Biology will treat the first patient in its clinical trial testing an induced pluripotent stem cell-based treatment for age-related macular degeneration.
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.
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.
An Orphan Disease Gets Adopted
Steve Bunk | Nov 12, 2000 | 7 min read
Courtesy of Ed Rowton, Walter Reed Army Institute of Research An anopheline mosquito taking a blood meal. Researchers and physicians, open your guides to rare diseases, for that may be the only place you'll encounter Jumping Frenchmen of Maine. There you also will find Kabuki Make-up Syndrome, Split-Hand Deformity, Stiff Person Syndrome, Tangier Disease, and Twin-Twin Transfusion Syndrome. These are among thousands of "orphan diseases," originally so called because they weren't "adopted" by spon

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