Drug Discovery World

JULY 18, 2023

For instance, virus-free approaches have been applied using CRISPR-Cas mediated homology-directed repair (HDR) to efficiently insert CARs into the TRAC locus of primary human T cells 7 , thereby improving their functionality. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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Disease DNA RNA Therapies 246

Codon

JULY 27, 2023

A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges. How do transcription factors couple up with the correct RNA molecule?

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Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges. How do transcription factors couple up with the correct RNA molecule?

Therapies 52

Therapies RNA DNA Engineering 52

Broad Institute

JUNE 27, 2024

They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration. CRISPRoff silences the targeted gene by adding methyl groups, chemical tags that prevent the gene from being transcribed or read into RNA and so from being expressed as protein.

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Disease Therapies Engineering Clinical Trials 142

Broad Institute

MAY 16, 2024

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.

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Therapies FDA Approval Engineering Disease 137

The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) has approved CABENUVA (consisting of Janssen’s rilpivirine and ViiV Healthcare’s cabotegravir), the first and only once-monthly, long-acting regimen for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in adults. 50 c/mL, meeting noninferiority criteria.

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FDA Approval Treatment RNA FDA 52

Codon

APRIL 2, 2023

Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). By combining this new variant with standard sequencing methods, it’s possible to identify RNA modifications in a really simple way. Nucleic Acids Research. Nature Biotechnology.

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DNA RNA Engineering Licensing 52

The Pharma Data

MARCH 20, 2021

Coronaviruses are RNA viruses and include common human coronaviruses, severe acute respiratory syndrome-CoV (SARS-CoV), Middle East respiratory syndrome-CoV (MERS-CoV) and SARS-CoV-2 ( 1 ). Detection of virus-specific antibodies allows for diagnosis of recent symptomatic or even asymptomatic infections. INTRODUCTION.

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Virus Assay Development Laboratories RNA 52

Broad Institute

DECEMBER 13, 2023

The studies are part of a package of 10 papers in Nature that take distinct, yet complementary, approaches to mapping the mouse nervous system at the single-cell level. The package also includes the spatial, single-cell atlas of the mouse brain and spinal cord that was led by Wang and Liu and was first published online in September in Nature.

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DNA Virus Engineering Research 140

The Pharma Data

OCTOBER 19, 2020

Spectrum’s self-contained saliva collection kit provides critical sample consistency while suspending and neutralizing viral RNA transcripts completely inactivating the live virus. Qualified commercial RNA extraction chemistries include Perkin Elmer, Thermo Fisher, Roche, and Qiagen. This press release features multimedia.

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RNA DNA Engineering Virus 52

Drug Discovery World

DECEMBER 15, 2022

Prime editing, while also employing a single strand nick, expands this scope to all 12 possible changes using a more complex prime editing guide RNA (pegRNA). DNA targeting specificity of RNA-guided Cas9 nucleases . Prime editing can be used for base swapping, as well as for targeted small insertions and deletions. References.

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DNA Clinical Trials Therapies RNA 130

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Read more in Nature Methods.

DNA 52

DNA RNA Engineering Vaccine 52

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Read more in Nature Methods.

DNA 52

DNA RNA Engineering Vaccine 52

The Pharma Data

NOVEMBER 30, 2020

Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that the primary efficacy analysis of the Phase 3 study of mRNA-1273 conducted on 196 cases confirms the high efficacy observed at the first interim analysis. “I

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Vaccine FDA Clinical Trials Disease 52

Codon

JUNE 26, 2023

A phage is a virus that infects bacteria. A T4 phage can hold 171,000 bases of DNA or other molecules, including proteins and RNA. For context, the AAV9 that is often used in gene therapies has a packaging limit of 4,700 DNA bases.) But LLMs will greatly increase the number of people who could make or distribute a virus.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

A phage is a virus that infects bacteria. A T4 phage can hold 171,000 bases of DNA or other molecules, including proteins and RNA. For context, the AAV9 that is often used in gene therapies has a packaging limit of 4,700 DNA bases.) But LLMs will greatly increase the number of people who could make or distribute a virus.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. If you want to edit a gene inside of a neuron, what do you do? These viruses enter the brain and deliver the gene-editing ingredients into neurons.

Vaccine 52

Vaccine DNA Virus Engineering 52

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. If you want to edit a gene inside of a neuron, what do you do? These viruses enter the brain and deliver the gene-editing ingredients into neurons.

Vaccine 52

Vaccine DNA Virus Engineering 52