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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.

Disease 144
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The science of ageing and restoring healthspan

Drug Target Review

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

Science 145
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The Biotech Startup Contraction Continues… And That’s A Good Thing

LifeSciVC

Beyond simply backing great science (separating the wheat from the chafe), setting a company up properly is critical, and early choices can get locked into the DNA of the company. Further, this lemming challenge has also moved well beyond oncology – think of all the “not-so-fast follower” autoimmune programs or metabolic disease stories.

DNA 109
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Marvin Caruthers receives inaugural Merkin Prize in ceremony at the Broad Institute for DNA synthesis technology

Broad Institute

Related links Merkin Prize Inaugural Merkin Prize in Biomedical Technology awarded to Dr. Marvin Caruthers for developing technology that efficiently synthesizes DNA The inaugural Richard N. Caruthers was announced as the winner in June for his development, in 1981, of an efficient, automated technology for synthesizing DNA.

DNA 52
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The power of combinations in blood cancers

Drug Target Review

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. 2012);12(4):237-251. 2 It is this complexity that necessitates powerful, targeted combination therapies. Front Pharmacol. 2018;9:1300. Published 2018 Nov 13.

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Driving Toward Nanopores

Codon

Biologists use nanopores for everything from diagnosing diseases and monitoring changes within rainforest ecosystems to discovering proteins from microbes frozen in Icelandic glaciers. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast. An Oxford Nanopore device.

DNA 77
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AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).