Broad Institute

JANUARY 8, 2024

In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA. Nature Biotechnology. Online January 8, 2024.

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Engineering Research Virus DNA 144

Drug Target Review

DECEMBER 6, 2023

Dear readers, RNA, or ribonucleic acid, plays a pivotal role in the intricate dance of cellular processes. We are committed to providing you with the highest quality content, and we believe that this report will deliver a different perspective on the topics covered.

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RNA DNA Vaccine Drug Development 52

Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. For both cancer and healthy genomes, we find that cysteine acquisition is a ubiquitous consequence of genetic variation that is further elevated in the context of decreased DNA repair.

Small Molecule 130

Small Molecule RNA DNA Therapies 130

Broad Institute

OCTOBER 23, 2024

In recent years, genetic editing technologies and other gene therapy approaches have given scientists the ability to alter the genes inside living cells. That is in part because of the ongoing challenge of understanding the DNA switches, called cis-regulatory elements (CREs), that control the expression and repression of genes.

Research 136

Research DNA Laboratories Molecular Biology 136

Drug Target Review

JULY 7, 2023

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

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DNA Engineering Therapies Regulations 105

Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?

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Therapies RNA Protein Expression DNA 59

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

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DNA RNA Disease Hospitals 128

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs.   The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach.

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Vaccine Treatment Clinical Trials Therapies 97

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

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Therapies DNA RNA Laboratories 52

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

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FDA Approval Treatment FDA DNA 97

DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Therapies DNA RNA Disease 52

Drug Target Review

APRIL 26, 2023

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs. The study appears in Nature Biotechnology.

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RNA Medical Schools Engineering DNA 98

Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays.

DNA 116

DNA Organic Chemistry Science Virus 116

Broad Institute

MARCH 22, 2024

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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Engineering Protein Production RNA Vaccine 140

Alta Sciences

FEBRUARY 19, 2025

The guidance ensures that these targeted evaluations help identify and mitigate safety concerns early in the development process, supporting the safe progression of oligonucleotide therapies into clinical trials. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

Drug Development 52

Drug Development Clinical Pharmacology Immune Response Drugs 52

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. Some fluoroquinolones — a class of antibiotics that inhibits DNA replication — may even cause psychosis.

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Vaccine Trials Therapies Treatment 96

Drug Target Review

NOVEMBER 13, 2023

The instructions to form a cell are encoded in DNA strands, wrapped in proteins and RNA, and wound together into a 3D structure called chromatin. Another finding was that regulatory T cells had distinct chromatin architecture features called DNA loops. This indicated other Foxp3 protein-containing complexes may be involved.

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Government DNA RNA Regulations 104

Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. approved a gene therapy for hemophilia A for the first time. They’re talking about a patient who has a rare genetic disease.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. approved a gene therapy for hemophilia A for the first time. They’re talking about a patient who has a rare genetic disease.

Therapies 52

Therapies RNA DNA Engineering 52

PLOS: DNA Science

APRIL 20, 2023

DNA is a sleek double helix, with “rungs” consisting of a purine base paired with a smaller pyrimidine base: adenine (A) with thymine (T) and guanine (G) with cytosine (C). . ” DNA encodes amino acid sequences comprising proteins, which impart traits. Watson and F.

DNA 52

DNA Virus Science RNA 52

Drug Target Review

SEPTEMBER 4, 2023

I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. 7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria.

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Drugs RNA DNA Disease 111

Codon

JUNE 11, 2024

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

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DNA Engineering RNA Small Molecule 83

PLOS: DNA Science

MARCH 14, 2024

Biotechnology has solved many problems, from recombinant DNA and monoclonal antibody-derived drugs, to gene therapy and stem cell transplants, to RNA-based vaccines and genetically modified plants that resist diseases and pesticides. Let Them Eat Snake appeared first on DNA Science. The post Cultivated Meat?

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DNA RNA Packaging Production 98

Broad Institute

DECEMBER 13, 2023

The team analyzed the full transcriptome of cells from nearly 100 regions across the mouse brain using high-throughput single-nucleus RNA sequencing, the preferred approach for efforts to create a human brain atlas. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs.

DNA 140

DNA Virus Research Engineering 140

Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. Support for $5 per month. Robinson A.O.

Therapies 52

Therapies RNA Engineering Vaccine 52

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., 2020-01-02.

Small Molecule 57

Small Molecule RNA DNA Disease 57

DrugBank

JULY 24, 2024

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases.

Small Molecule 98

Small Molecule Drugs Clinical Trials Therapies 98

Fierce BioTech

NOVEMBER 16, 2023

Beyond cfDNA: Harnessing the Power of Exosomes to Improve Sensitivity for Cancer Mutation Detection Liquid biopsy markers are advantageous because they can be used to assess overall prognosis, response to therapy, and likelihood of relapse or progression. Download now. Download now.

RNA 52

RNA DNA Therapies Treatment 52

Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Research Immune Response Clinical Trials Therapies 137

The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma.

Treatment 52

Treatment RNA DNA Science 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

Therapies 40

Therapies RNA Licensing Licensing 40

PLOS: DNA Science

MAY 25, 2023

In these 50 or so conditions, symptoms may appear earlier and worsen from generation to generation, as the mutant gene grows, adding copies of a 3- or 4-base DNA sequence. Now Avidity Biosciences has developed a candidate drug that weds a monoclonal antibody (MAb) to a short piece of RNA – a small interfering RNA (siRNA).

Treatment 52

Treatment DNA RNA Clinical Trials 52

Codon

APRIL 2, 2023

But now, by studying DNA extracted from microbes in the blood of almost 10,000 healthy people, this paper shows that there is no such thing. Read Transcription factors bind to DNA and control gene expression. Read Switchable hydrophobic pockets in DNA protocells enhance chemical conversion. Nature Microbiology. Meeussen J.V.W.

DNA 52

DNA RNA Engineering Licensing 52

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

Therapies 52

Therapies Disease Medical Schools Virus 52

Drug Target Review

AUGUST 9, 2023

The specific proteins found in these aggregates can vary, but approximately 95 percent of ALS patients and 50 percent of FTD patients develop aggregates containing the TDP-43 protein (short for tar DNA binding protein 43). 14 TDP-43 is a DNA and RNA binding protein that regulates the expression and splicing of several target transcripts.

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RNA Regulations Disease DNA 98

Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

Therapies 52

Therapies Laboratories Molecular Biology RNA 52

Codon

SEPTEMBER 29, 2024

But, regardless of which was first, they all operated with the same core data as their mechanism for understanding life: messenger RNA ( mRNA ). It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life.

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