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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. “As
Estrogen receptor (ER)-negative breast cancers are known to be aggressive and unresponsive to anti-estrogen therapy, and triple negative breast cancers are associated with poor prognosis and metastasis. Thus, new targeted therapies are needed.
Curcumin modulates the gene and proteinexpression levels of ferroptosis mediators via JNK signaling. Ferroptosis, a new form of regulated cell death, plays a vital role in the pathogenesis and therapy of cancers.
We previously showed that epigenetic histone dysregulations were associated with decreased Brain Derived Neurotrophic Factor (BDNF) expression in a GWI rat model. GWI has no effective therapies. Ketamine (KET) has recently been approved by the FDA for therapy-resistant depression. mg/kg s.c.,
Linagliptin administered to the type 1 diabetic mouse heart significantly reduced the expression levels of the total and cleaved forms of ATF6, ATF4, and p-JNK, caspase 3. According to ELISA findings, TUDCA was more effective in reducing NOX 1 and MDA levels than linagliptin.
Secondly, circRNAs can be engineered for more efficient proteinexpression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. There are three major reasons for why Circio has selected AATD as its lead gene therapy program.
In addition, western blot analysis showed that CA-COS inhibits the proteinexpression of iNOS and nuclear factor kappa B (NF-kB), including p50 and p65, and mitogen-activated protein kinase (MAPK) signaling pathways.
The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.
This interview explores how CoRegen’s innovative techniques are not only transforming cancer treatment but also hold promise for a wide range of medical applications, all while minimising the side effects often associated with traditional therapies. What are the key benefits of your approach?
Broad expression of FAP demonstrated in tumors or in tumor stroma across many solid tumors 1 ,2 ,3. Novartis Oncology continues to reimagine cancer care through development of robust radioligand therapy portfolio. We continue to invest in radioligand therapy as one of the four unique platforms of Novartis Oncology.
For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.
” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. AI + Bio *Transfer learning for cross-context prediction of proteinexpression from 5’UTR sequence. Molecular Therapy. Gene Therapy. McGrady N.R.
Etta Biotech”), to set up a high titer transient proteinexpression platform for high quality protein production using JS Bio’s transient transfection media. JS Bio becomes the exclusive cell culture supplier for Etta Biotech’s transient transfection high titer proteinexpression platform. About JS Bio.
A pivotal moment for me was when I realised that if I understood disease mechanisms on a molecular level, I could be part of an industry that helps develop medicines/therapies for those who need them most. I loved learning how to explain at the molecular level what was happening in nature, in human beings and disease settings.
Based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a significant improvement in both progression-free survival and overall survival Enhertu has now been granted five Breakthrough Therapy Designations, including three in breast cancer and one in both lung and gastric cancers.
Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness. Fortunately, personalized treatment plans help circumvent therapy resistance while optimizing response rate and improving quality of life.
As discussed above, recent advances in gene editing tools and transposon-based vectors have facilitated targeted and consistent cell line engineering for the development of proteinexpressing CHO cell lines. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014. Biotechnol Prog. 2017 Nov;33(6):1468-1475.
Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.
For instance, a dataset generated to study proteinexpression in one context might also reveal valuable information about other biological pathways or processes. Additionally, exploratory analyses can be valuable for identifying new biological markers or hypotheses.
Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify proteinexpression or use cells to alter/restore a specific cell type.
Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.
10 Reported cAMP as the second messenger signalling molecule conserved from bacteria to humans and modulates several biological processes, including proteinexpression, gene transcription, and cell development and differentiation.
Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021.
FBX-101 is a first-in-human AAV gene therapy. Harpoon Therapeutics dosed the first patient with HPN328 in its Phase I/II trial of small cell lung cancer (SCLC) and other tumors associated with DLL3 expression. The study hit the mark on its primary biological endpoint of micro-dystrophin proteinexpression.
The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to 6 weeks aged and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne dystrophy (DMD).
(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based proteinexpression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,
The target of DF7001 is 5T4, a proteinexpressed on cancer cells and stromal cells that support tumor growth associated with poor prognosis in several cancers, including non-small cell lung cancer (NSCLC), pancreatic cancer, breast cancer, and head and neck squamous cell carcinomas (HNSCC). View the full release here: [link].
The vaccine candidate, presently at a preclinical development stage, is a non-chemically modified mRNA, encoding the prefusion stabilized full-length spike protein of the SARS-CoV-2 virus, and formulated within Lipid Nanoparticles (LNPs). CureVac had its initial public offering on the New York Nasdaq in August 2020.
Tecentriq also has four approved indications in NSCLC as either a single agent or in combination with targeted therapies and/or chemotherapies. It was the first approved cancer immunotherapy for first-line treatment of adults with extensive-stage small cell lung cancer (SCLC) in combination with carboplatin and etoposide (chemotherapy).
3,4,5 TROP2 is a proteinexpressed in more than 90% of NSCLC tumors. Rahway, NJ, USA), an anti-PD-1 therapy. 3,4,5 TROP2 is a proteinexpressed in more than 90% of NSCLC tumors. Rahway, NJ, USA), an anti-PD-1 therapy. More than one million people are diagnosed with NSCLC at an advanced stage each year.
A cure for sickle cell anaemia A recent scientific breakthrough in the treatment of sickle cell anaemia —a genetic disorder marked by episodes of intense pain—illustrates how combining GWAS with cutting-edge molecular tools like gene editing can identify causal variants and lead to innovative therapies.
In Europe, Tecentriq now has four approved indications in NSCLC, including as a single agent or in combination with targeted therapies and/or chemotherapies. Tecentriq has shown clinically meaningful benefit in various types of lung cancer, with five currently approved indications in markets around the world.
Basel, 11 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data that show OCREVUS® (ocrelizumab) is a highly effective treatment option for people with relapsing-remitting multiple sclerosis (RRMS) who experienced a suboptimal response to their prior disease modifying therapy (DMT). and primary progressive MS (PPMS).
(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based proteinexpression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,
This investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi, the bacteria that cause Lyme disease. OspA is one of the most dominant surface proteinsexpressed by the bacteria when present in a tick.
Enhertu previously received Priority Review and Breakthrough Therapy Designation (BTD) in the U.S. 3 HER2 is a tyrosine kinase receptor growth-promoting proteinexpressed on the surface of many types of tumors including breast, gastric, lung and colorectal cancer. Targeted Therapies for Stomach Cancer. 2,5 In the U.S.,
This investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi , the bacteria that cause Lyme disease. OspA is one of the most dominant surface proteinsexpressed by the bacteria when present in a tick.
In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no proteinexpression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A proteinexpression in neurons results in abnormal communications between neurons.
For example, advances in media and feeds have contributed to higher cell proteinexpression by optimising nutrient availability and reducing accumulation of waste products within a culture. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.
Dr. Lederman continued, “Despite the recognized promise of anti-CD40-ligand mAb therapy, first generation anti-CD40-ligand mAbs were limited because their crystallizable fragment (Fc) domain interacted with a cell surface receptor called Fc?RII, RII, which resulted in an increased risk of thrombosis. International Immunol. (11):1583
In addition, over 170,000 people have been treated with OCREVUS, our first-in-class B-cell therapy, and we are incorporating years of clinical trial data and real-world evidence to optimise its potential to improve outcomes for patients with MS.”. Roche’s Chief Medical Officer and Head of Global Product Development. “In
Effect of Celecoxib vs Placebo as Adjuvant Therapy on Disease-Free Survival Among Patients With Breast Cancer: The REACT Randomized Clinical Trial Published in JAMA Oncology This large (n=2639) trial of adjuvant celecoxib vs. placebo in women with HER2-negative primary breast cancer finds no effect on disease free survival at 10-years follow up.
An initial analysis of data from 17/22 patients who completed one of these assessments showed substantially decreased neurocognitive abilities compared to children of the same age level despite the use of multiple anti-epileptic therapies. .–( BUSINESS WIRE )– Stoke Therapeutics , Inc. STK-001 is designed to upregulate Na V 1.1
This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications.
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