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tag rare diseases immunology disease medicine neuroscience

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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.
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. 
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.
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.
Blue T cell with other blurred T cells in the background
Woman Seemingly Cured of HIV After Umbilical Cord Transplant
Natalia Mesa, PhD | Feb 16, 2022 | 3 min read
Umbilical cord blood may be a good alternative to bone marrow transplants for treating HIV in patients with HIV and cancer.
Journal club logo on purple background
The Scientist's Journal Club: Transcriptomics
The Scientist | Sep 20, 2023 | 2 min read
Scientists discuss their latest findings on immune cell dynamics, neurodegenerative disease risk factors, and rare cell types obtained from bulk and single cell RNA sequencing experiments.
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.
neuron surrounded by immune cells
Meningitis Bacteria Trigger Headaches, Then Sneak Into the Brain
Natalia Mesa, PhD | Mar 6, 2023 | 3 min read
Researchers find that bacteria stimulate a headache-causing pain pathway to suppress the immune system and infect the brain. 
Image of a white cardboard box with a blue vertical stripe on the left side, the word “Apixaban” in blue lettering at the bottom, and a depiction of apixaban’s molecular structure in black.
Blood Thinner Ineffective for COVID-19 Patients: Study
Dan Robitzski | Nov 28, 2022 | 2 min read
A clinical trial finds that the anticoagulant apixaban, which has been prescribed to help COVID-19 patients recover, is ineffective and in rare instances dangerous.

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