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tag rare diseases disease medicine neuroscience cell molecular biology

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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. 
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.
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.
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.
Tiled blue-gray MRI readouts of a human brain.
Cancer Tied to Reduced Risk of Alzheimer’s Disease
Dan Robitzski | Apr 14, 2022 | 7 min read
Observational evidence for the connection is solidifying, and some clues are emerging about the mechanisms that may explain it.
Glowing red DNA on bluish background
Redesigning Medicine Using Synthetic Biology
Alison Halliday, PhD, Technology Networks | Jun 21, 2023 | 5 min read
Drawing inspiration from nature, synthetic biology offers exciting opportunities to transform the future of medicine.
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.
3D illustration of damaged myelin sheath seen in demyelinating diseases such as multiple sclerosis.
Tracking Down Innate Immune Cells in Multiple Sclerosis
Mariella Bodemeier Loayza Careaga, PhD | Dec 4, 2023 | 5 min read
A novel PET tracer targeting a receptor in myeloid cells can help monitor disease progression in a mouse model of multiple sclerosis.
Different colored cartoon viruses entering holes in a cartoon of a human brain.
A Journey Into the Brain
Danielle Gerhard, PhD | Mar 22, 2024 | 10+ min read
With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy.

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