Drug Target Review

DECEMBER 5, 2024

2 Structure and function of AAV capsids in gene therapy Wild-type AAV is composed of a protein shell (capsid) that contains a single-stranded DNA genome encoding proteins involved in viral replication, structure and assembly. Adeno-associated virus as a delivery vector for gene therapy of human diseases. Labcompare. 2024;9(1):78.

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Drug Target Review

SEPTEMBER 8, 2023

The cell’s nucleus, containing compacted DNA, is centrally positioned within the cell. It didn’t have the tools that are important for nuclear receptors, namely the ability to interact with DNA and the ability to translocate from the cytoplasm to the nucleus,” says Cheroutre.

Regulations 98

Regulations Research DNA Immune Response 98

The Pharma Data

JANUARY 18, 2021

Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy. WUHAN, China and NEWARK, DE. , a subsidiary of Wuhan Neurophth Biotechnology Ltd.

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Therapies Immune Response DNA Treatment 52

The Pharma Data

JANUARY 13, 2021

The vaccine produced an immune response of all 805 clinical trial participants within two months of inoculation, according to results published Jan. The new vaccine is made up of a deactivated cold virus into which scientists cut-and-paste a genetic version of the “spike” protein used by the coronavirus to infect cells. .”

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Vaccine Trials Clinical Trials Immune Response 40

The Pharma Data

OCTOBER 14, 2020

(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic. About TNX-1800.

Vaccine 40

Vaccine Pharmaceuticals Small Molecule Virus 40

Broad Institute

JUNE 27, 2024

Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration.

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Drug Target Review

JANUARY 15, 2024

Each T cell expresses a TCR specific for an HLA‑presented peptide signal, and when that signal is detected a T cell expressing the relevant TCR can launch a complex array of immune effector responses that can suppress or destroy tumours or virus-infected cells. Many potential candidate antigens are under consideration.

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Engineering DNA Virus Medical Schools 110

Codon

FEBRUARY 13, 2024

doi: 10.2210/rcsb_pdb/goodsell-gallery-048 The Virus that Cures It’s been over 25 years since the science magazine Discover first ran an extraordinary article about how a long-forgotten medical treatment, used in the former Soviet country of Georgia, could save us from the growing threat of untreatable, drug-resistant infections.

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Therapies Virus DNA Clinical Trials 137

Drug Target Review

OCTOBER 26, 2023

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. Engineering adeno-associated virus vectors for gene therapy.

Engineering 105

Engineering Virus Therapies Immune Response 105

Broad Institute

AUGUST 11, 2022

Microbial armory In an earlier study, the researchers scanned data on the DNA sequences of hundreds of thousands of bacteria and archaea, which revealed several thousand genes harboring signatures of microbial defense. The cells underwent a dramatic defensive response and survived. The finding was striking and unprecedented.

DNA 52

DNA Virus Immune Response Bioinformatics 52

PLOS: DNA Science

OCTOBER 12, 2023

Can they deliver healing genes without triggering an overactive immune response? The details are disturbingly reminiscent of the famous case of Jesse Gelsinger , who died from a ferocious immune response to experimental gene therapy in September 1999. million DNA bases. Two other design strategies add precision.

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Therapies Immune Response DNA Virus 69

Broad Institute

AUGUST 7, 2023

Related people Paul Blainey Nir Hacohen A signaling protein known as STING is a critical player in the human immune system, detecting signs of danger within cells and then activating a variety of defense mechanisms.

Regulations 91

Regulations Clinical Trials Engineering Science 91

Drug Target Review

OCTOBER 2, 2024

While commonly used, AAVs have limitations, including immune reactions and difficulties with re-dosing due to pre-existing antibodies. As with all CGTs, safety concerns, particularly related to immune responses and long-term effects, are crucial in cardiology. Allogeneic cell therapies must avoid immune rejection.

Therapies 98

Therapies Disease RNA Engineering 98

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. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain. From Stahl E.C.

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. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain. From Stahl E.C.

Vaccine 52

Vaccine DNA Virus Engineering 52

PLOS: DNA Science

JANUARY 4, 2024

.” CRISPRs are short DNA sequences, peppered with repeats, that latch onto DNA-cutting enzymes, commandeering and directing them to snip certain parts of a chromosome. The microbes deploy them to dismantle the genetic material of infecting viruses, a little like an immune response.

FDA Approval 52

FDA Approval DNA Therapies Clinical Trials 52

DrugBank

FEBRUARY 26, 2025

Targeting Novel Bacterial Pathways Traditional antibiotics often target essential bacterial processes such as cell wall synthesis or DNA replication. Immunomodulatory therapies, which enhance the host immune response to infections, are also being investigated as adjunctive treatments.

Therapies 52

Therapies Pharmaceutical Companies Pharmacokinetics Treatment 52

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” a lower risk of immune responses.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” a lower risk of immune responses.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

APRIL 30, 2023

Yeast die for two reasons: Either their nucleolus (where the DNA is kept) degrades and dies, or their mitochondria whimpers out and they stop making energy. The vaccine printer can make lots of different types of vaccines, including protein, DNA, and mRNA ones, but I’m sure this is all quite expensive right now. From Zhang et al.

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Vaccine DNA Engineering RNA 52

Codon

APRIL 30, 2023

Yeast die for two reasons: Either their nucleolus (where the DNA is kept) degrades and dies, or their mitochondria whimpers out and they stop making energy. The vaccine printer can make lots of different types of vaccines, including protein, DNA, and mRNA ones, but I’m sure this is all quite expensive right now. From Zhang et al.

Vaccine 52

Vaccine DNA Engineering RNA 52

Drug Target Review

DECEMBER 28, 2023

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell. Neoantigens are recognised as non-self and trigger an immune response.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

The Pharma Data

NOVEMBER 29, 2020

Compared to placebo, participants vaccinated with three HB-101 doses had: a 48 percent reduction in CMV viremia (presence of CMV DNA in the blood); a 42 percent reduction in the use of antiviral therapy; and. 3 The majority of CMV infections are not serious, and the virus can lay dormant in the body for years.

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Vaccine Clinical Trials Disease Therapies 40

Codon

SEPTEMBER 29, 2024

mRNA was the intermediate stage between DNA and protein, a dynamic entity that shifted depending on the second-to-second needs of the cell, able to point out if a cell was cancerous or stressed, what kind of cell it was, and so on. Collections of mRNA have been understood as a proxy for cell states for decades.

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Engineering Virus DNA Therapies 145

Codon

APRIL 9, 2023

Thousands of transcription factors — proteins that bind DNA and control gene expression — were studied in human cells. Read DNA Sequencing & Synthesis *Quantitative analysis of tRNA abundance and modifications by nanopore RNA sequencing. Bergs A.C.F. Nature Communications. Porta-de-la-Riva M. Nature Methods.

Therapies 52

Therapies RNA Engineering Vaccine 52

Codon

AUGUST 27, 2024

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Reading DNA The first step in detecting a novel pathogen is recognizing it as an anomaly amidst a noisy background of other material. After copying the DNA to form a big pool, each piece is sequenced.

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DNA Laboratories Hospitals Virus 112

PLOS: DNA Science

MAY 16, 2024

Many of the links below are to some of the 100+ DNA Science posts that I wrote during the pandemic. A Novel Virus Appears In retrospect, everything unfolded with astonishing speed. Whenever and wherever the virus emerged, CDC officially alerted clinicians to the new, deadly respiratory illness on January 8, 2020.

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Virus DNA RNA Vaccine 97

The Pharma Data

DECEMBER 27, 2020

It is a selective modulator of neuropilin-2 that downregulates the innate and adaptive immune response in inflammatory diseases. Transgene and BioInvent International received regulatory approval in Belgium for a Phase I/IIa trial of oncolytic Vaccinia virus BT-001. platforms and the human GM-CSF cytokine. Most Read Today.

Clinical Trials 52

Clinical Trials Trials Therapies Virus 52

Codon

DECEMBER 12, 2024

It would also be undetectable by key parts of the human immune system. Some scientists even think it had a virus defense system; “researchers say LUCA likely housed 19 CRISPR genes, which bacteria use to slice up viral threats,” reports Quanta Magazine. 4 As far as we know, right-handed proteins never occur naturally.

RNA 105

RNA DNA Vaccine Virus 105

Codon

APRIL 16, 2023

Skin microbes can trigger strong immune responses. These microbes were engineered to express tumor antigens that could “elicit T cells that were licensed by the commensal immune program but specific for a tumor,” including both CD4+ and CD8+ T cells, according to the study. coli DNA using a Retro-Cascorder.

Engineering 52

Engineering DNA RNA Science 52

The Pharma Data

DECEMBER 20, 2020

It is an optimized, non-chemically modified mRNA that codes for the prefusion stabilized full-length spike protein of the SARS-CoV-2 virus. Pfizer and BioNTech announced additional data on neutralizing antibody and T-cell responses from their Phase I/II trial of their COVID-19 vaccine conducted in Germany.

Vaccine 52

Vaccine Trials Therapies Small Molecule 52

The Pharma Data

JANUARY 21, 2021

The test valuates mitochondrial DNA levels. When mitochondrial DNA, normally found inside cells, in the mitochondria, spills into the bloodstream, it signals that a particular type of violent cell death is occurring. They measured the patients’ mitochondrial DNA levels on the first day of their hospital stays. found in the U.K.

Research 52

Research DNA Vaccine Immune Response 52

Codon

MARCH 26, 2023

Link A DNA tumor virus globally reprograms host 3D genome architecture to achieve immortal growth. Link DNA storage in thermoresponsive microcapsules for repeated random multiplexed data access. Link Semi-rational evolution of a recombinant DNA polymerase for modified nucleotide incorporation efficiency. Antika T.R.

DNA 52

DNA Engineering RNA Science 52

PLOS: DNA Science

JULY 25, 2024

A virus that directly attacks the immune response presents a special challenge. Dr. Fauci explains the nomenclature of the virus – combinations of 18 hemagglutinin (H) and 11 neuraminidase (N) proteins that comprise the surface – and the chain of infection from birds to mammals. Ebola virus fever is horrific.

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Virus Science Vaccine Government 94

PLOS: DNA Science

FEBRUARY 20, 2025

Influenza incidence and severity are increasing , against a backdrop of novel drifts (small genetic changes) and shifts (larger changes) to the virus, all against a backdrop of novel infection pathways, such as birds to cows to people. The mRNA in COVID vaccines tells cells to produce the virus’s spike protein.

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Vaccine Science RNA Disease 91

PLOS: DNA Science

MAY 22, 2025

They used a highly precise form of CRISPR gene editing to correct a mutation – swapping out one DNA base for another – that lay behind the boy’s inability to break down proteins in food. In a gene therapy trial for an immune deficiency, such an effect caused leukemia in a few boys.

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Therapies DNA Clinical Trials Hospitals 65

PLOS: DNA Science

JULY 10, 2025

“Fluoride replaces human DNA with hippo DNA, but only where directly applied.” Adenovirus, rotavirus, hepatitis A virus, noroviruses, coxsackievirus, echovirus, reovirus, and astrovirus 4. The innate immune response and the adaptive immune response b. “Fluoride is a known teratogen.

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Immune Response DNA RNA Vaccine 76