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tag rare diseases disease medicine

Abstract illustration depicting coronavirus research concept.
Curiosity and Compassion Fuel Rare Disease Research
The Scientist Staff | Jan 1, 2024 | 1 min read
Lauren Drouin shares how personal connections and scientific curiosities drive her work on gene therapy viral vectors. 
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.
Healthcare and medicine, Doctor touch and diagnose a virtual Human Lungs with Covid-19 or coronavirus spread inside on modern interface screen on laboratory, Innovation and Medical technology.
Uncovering Rare Disease Genetic Pathways with Global Biobanks
Deanna MacNeil, PhD | Mar 20, 2023 | 3 min read
Researchers discover new idiopathic pulmonary fibrosis risk factors with multi-ancestry analyses that increase representation of understudied populations.
An excited-looking toddler crawls towards toys while her mother watches on.
Doctors Treat a Rare Genetic Condition Before Patient Is Born
Dan Robitzski | Nov 10, 2022 | 2 min read
Thanks to continued weekly medications, a 16-month-old girl shows no symptoms of a severe genetic disease that typically kills children before they turn two.
Photo of Rosa Bacchetta, associate professor of pediatrics at Stanford University
The Future of Gene Therapy for a Rare Pediatric Autoimmune Disease
Niki Spahich, PhD | Jul 10, 2023 | 3 min read
By editing a mutated immune regulatory gene in patient cells, Rosa Bacchetta brings hope to those suffering from IPEX syndrome.
Haydeh Payami is wearing a purple dress and an orange and pink scarf and standing in front of a whiteboard.
A Microbial Link to Parkinson’s Disease
Mariella Bodemeier Loayza Careaga, PhD | Dec 4, 2023 | 6 min read
Haydeh Payami helped uncover the genetic basis of Parkinson’s disease. Now, she hopes to find new ways to treat the disease by studying the gut microbiome.
A false color transmission electron microscope micrograph showing the nuclear envelope, the rough endoplasmic reticulum, the nucleus, and the cytoplasm.
New Gene Mutants Identified in Rare Motor Neuron Diseases
Clare Watson | Oct 17, 2022 | 2 min read
The discovery of gene variants in cases of hereditary spastic dysplasia could provide a diagnosis to affected families where no genetic cause could be found before.
Ribbon diagram of the protein coat of an adeno-associated virus
Preprints Propose Constellation of Causes for Kids’ Liver Disease
Christie Wilcox, PhD | Jul 25, 2022 | 2 min read
Two independent groups suggest the suite of recent unexplained hepatitis cases may stem from coinfection with an adeno-associated virus and a helper adeno- or herpesvirus, a duo which may be especially virulent in children with a particular genetic variant.
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.

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