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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.
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.
Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.
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.
Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.
There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Because mRNA is not a virus, it doesn’t have to meet biosafety requirements for manufacturing and administration that an AAV approach would need.
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.
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.
Signal Transduction and Targeted Therapy. Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Ribozyme-mediated RNA synthesis and replication in a model Hadean microenvironment. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding.
To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.
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. In their study, the researchers focused on a variant of an engineered recombinant adeno-associated virus (rAAV) known as PHP.eB.
” — 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. Molecular Therapy. Support for $5 per month.
But what happens if you restore these cone cells, using gene therapy? 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.
But what happens if you restore these cone cells, using gene therapy? 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.
Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.
Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.
Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. We discovered it is highly effective and environmentally friendly.
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.
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?
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.
He has also emerged in recent years as a skilled and relatable science communicator, renowned for his efforts at Broad and beyond during the COVID-19 pandemic to unpack the fast-moving research on the new virus in an accessible way and give practical advice about masking, vaccines, and other public health measures.
The COVID-19 pandemic has highlighted the urgent need for a novel, oral antiviral to treat this highly infectious and often deadly virus,” said Jean-Pierre Sommadossi, Ph.D., “Roche shares our passion for delivering innovative new medicines to address great unmet medical needs. Chief Executive Officer and Founder of Atea Pharmaceuticals.
A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.
Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNAvirus that has a trimeric spike (S) protein on its viral envelope. FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. About Dynavax.
The study was published online in advance in the journal Molecular Therapy – Nucleic Acids and is now available in the final version. The virus carries the information to produce a series of proteins, capable of inhibiting antiviral recognition systems of the infected cell. Camouflage protects virus from immune system.
During challenge with the original SARS-CoV-2 virus, animals vaccinated with CV2CoV were found to be better protected based on highly effective clearance of the virus in the lungs and nasal passages. Clearance of the virus in the lungs and nasal passages of the animals was tested following challenge infection with the original virus.
Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNAvirus that has a trimeric spike (S) protein on its viral envelope. Additionally, Clover is leveraging its in-house GMP biomanufacturing capabilities to support large-scale production of its biologic therapies.
For security reasons, at the time COVID-19 had to be grown in BSL-3 labs, which can be dangerous because they were handling the live virus. These are synthetic, not the live virus, so do not pose a risk of infection. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays.
” Dr. Richard Hatchett , Chief Executive Officer of CEPI, commented: “To end the acute phase of this pandemic, and to control the virus in the longer term, the world needs multiple safe and effective vaccines which can be deployed in a range of populations and countries. About CpG 1018.
The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.
That’s how easy it’s for SARS-CoV-2, the virus that causes COVID-19, to enter your nose. “We used NanoSTING because the adjuvant for intranasal vaccination and single-cell RNA-sequencing to verify the nasal-associated lymphatic tissue as an inductive site upon vaccination. Breathe in, exhale. But now, we’re one step closer.
The once-a-month regimen was preferred by nine out of 10 patients over the daily oral therapy. While much more remains to be done to make HIV history, today’s milestone reminds us how far medical innovation has come since the first reported cases of the virus almost 40 years ago.”. clinical practices. Most Read Today. Source link.
The outcome confirms that only one single case was attributable to the original SARS-CoV-2 virus. “In addition, the variant-rich environment underlines the importance of developing next-generation vaccines as new virus variants continue to emerge.” More than half of the cases (57%) were caused by Variants of Concern.
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. Gene Therapy. Nucleic Acids Research.
5 of its ongoing Phase 2 extension study, Study 211, examining vebicorvir (VBR, or ABI-H0731) in patients with hepatitis B virus infection. The company’s other two Phase 2 trials, Study 201 and 202, showed that the addition of VBR to NrtI therapy achieved a quicker, deeper level of viral suppression than with NrtI alone.
In addition to sequencing patients, the researchers looked for antibodies to the HTLV-1 virus in the patients’ spinal fluid. We each have two DNA copies of the KIF1A gene; each copy is called an allele and makes its own RNA, which is then translated into protein. What is an allele specific ASO?
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. Molecular Therapy.
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. Molecular Therapy.
By testing only what’s medically relevant and necessary, we enable targeted therapy, avoid misuse of antibiotics and reduce unnecessary intervention.”. 1-3 tract infections include both upper tract infections, just like the cold , and lower tract infections, like pneumonia, bronchitis and tuberculosis.
. “There remains an urgent need for SARS-CoV-2 therapeutic agents that target parts of the virus other than the spike protein that are not as likely to evolve.” Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro. ” The findings published today (Feb. 25) in the journal Communications Biology. .
The study showed the antiviral activity of GSK’254, establishing a relationship between dose and antiviral response, with the 140 mg and 200 mg doses showing the greatest reduction in plasma HIV-1 RNA. These findings were presented today at the (virtual) 2021 Conference on Retroviruses and Opportunistic Infections (CROI). log 10 and -2.0
During the pandemic, Pfizer will offer its oral therapy through a tiered pricing approach, pending country authorization or approval, based on the income level of each country to promote equity of access across the globe. High and upper-middle income countries will pay more than lower income countries.
. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,
Immunization of non-human primates (rhesus macaques) with BNT162b2, a nucleoside-modified messenger RNA (modRNA) candidate that expresses the SARS-CoV-2 spike glycoprotein, resulted in strong anti-viral effects against an infectious SARS-CoV-2 challenge. billion doses by the end of 2021.
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