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tag gene therapy evolution culture disease medicine

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.
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. 
Infusion of Artificial Intelligence in Biology
Meenakshi Prabhune, PhD | Feb 23, 2024 | 10 min read
With deep learning methods revolutionizing life sciences, researchers bet on de novo proteins and cell mapping models to deliver customized precision medicines.
Gene Therapy Targets Canavan Disease
Douglas Steinberg | Sep 16, 2001 | 6 min read
The Canavan trial signals a new phase in a 10-year offensive that gene therapy researchers have waged against neurodegenerative disorders.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019 | 3 min read
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
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.
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.
A bat flying in a dark cave
Turning on the Bat Signal
Hannah Thomasy, PhD | Mar 15, 2024 | 10+ min read
Scientists around the world investigate how bat immune systems cope with viral attacks and how this information could be used to keep humans safe.
A person moving the hands of a vintage clock backwards.
Synthetic Circuits Reveal the Key to Rewinding the Cellular Clock
Charlene Lancaster, PhD | Mar 12, 2024 | 4 min read
Using a circuit-based system, scientists determined the ideal transcription factor levels to promote the successful reprogramming of fibroblasts into induced pluripotent stem cells.
Gene Therapy Coming of Age?
Dan Cossins | Jul 11, 2013 | 4 min read
Using lentiviral vectors to replace mutated genes in blood stem cells, scientists successfully treat two rare diseases apparently without causing harmful side effects.

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