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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. By Sarah C.P. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

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The role of CRISPR in microbiome engineering breakthroughs

Drug Target Review

Using a synthetic guide RNA (gRNA), scientists can target a specific DNA sequence and employ Cas9 to cut it. Today, however, we will explore a different application of CRISPR: microbiome engineering… Clustered regularly interspaced short palindromic repeats (CRISPR) was first discovered in the genome of marine bacteria.

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Messenger RNAs with multiple “tails” could lead to more effective therapeutics

Broad Institute

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

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Evolved prime editors are smaller and more efficient for therapeutic applications

Broad Institute

Now researchers at the Broad Institute of MIT and Harvard have used cutting-edge continuous laboratory evolution and engineering methods to develop improved versions of the gene-editing tool. Reverse transcriptase proteins that copy RNA templates into strands of DNA are found naturally in all plant and animal cells and in many viruses.

DNA 145
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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor. This engineered capsid opens up a world of possibilities."

Therapies 137
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Day Zero Antivirals for Future Pandemics

Codon

When COVID-19 emerged in 2019, by contrast, mRNA vaccines developed by Pfizer and Moderna took just 326 days from the initial sequencing of the virus to gaining approval for emergency use. These drugs would be ideal tools to bridge the wait for a vaccine against a quickly-spreading virus.

RNA 85
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

To deliver CHARM throughout the brain, the team collaborated with scientists in the lab of Ben Deverman , senior director of vector engineering at the Broad and a co-author of the study. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration.

Disease 142