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Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies.
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.
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.
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.
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. In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. Neoantigens are recognised as non-self and trigger an immuneresponse.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.
His mother had a presentation of the disease that suggested her immune system was already on the job. 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.
HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immuneresponses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immuneresponses and facilitating antibody production.
2 In response, DNA-damaging agents that could target the entire cell cycle received renewed attention as ADC payloads. More recently, researchers have begun to look at conjugating immunomodulatory agents to antibodies to directly activate an immuneresponse against the tumour. Fu Y, Ho M. 1 , 33–43 (2018).
The molecular tools of CRISPR were borrowed and developed from the natural immuneresponse of bacteria to viruses – bacteriophages – that infect them. CRISPR may make sense for replacing a single mutant gene, like the one behind sickle cell disease, for which a CRISPR-based gene therapy was recently approved.
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.
Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.
We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. Generally, IO has been focused on harnessing the anti-tumour activity of certain cancer-fighting T-cells , a key cell type involved in the adaptive immune defense system.
Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 9 In addition, host immuneresponses further add to the complexity of developing cell-specific AAV capsids for clinical applications. Molecular Therapy 20 , 1831-1832 (2012).
2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).
Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy.
Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immuneresponses and cost.
gene therapy company (hereafter Neurophth), and AAVnerGene Inc. ,
Unlike almost every other cell type (except B cells), T cells do not have the exact same chromosomal DNA sequences as other cells in the body. To be therapeutically useful, antigenic peptides must be presented in a way that allows immuneresponses to destroy cancer cells without causing unacceptable damage to healthy tissue.
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. Of particular interest was how genetic therapies were delivered.
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Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.”
About INOVIO’s DNA Medicines Platform.
mg and 2.0
Their cone cells, which are responsible for color vision, don’t work like normal. 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. LEGO robot to make sucrose gradients.
Their cone cells, which are responsible for color vision, don’t work like normal. 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. LEGO robot to make sucrose gradients.
Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. To achieve this, scientists utilise various technologies that function similarly to scissors, precisely cutting the DNA at a particular location.
Since cancer cells are under chronic stimulation of ER stress, the polypeptides cannot activate innate immune sensors in cancer cells even upon the polypeptide treatment. Which natural agonists impede the activation of intracellular DNA sensors in APCs, and what challenges do they pose?
Antibody-Drug Conjugates: Advancing Precision Therapy ADCs represent a compelling combination of targeted antibody therapy and potent chemotherapy, aiming to enhance treatment efficacy while minimizing systemic toxicity. Currently, there are 12 approved ADC drugs, and several hundred more are in the development pipeline.
The idea to redirect an immuneresponse against a pathogen to fight cancer goes back to 1891, when Manhattan bone surgeon William Coley became intrigued by a man’s neck tumor that melted away after he contracted a nasty Streptococcus skin infection. For many patients, BCG stops the cancer.
Leadership in DNA damage response demonstrated in multiple presentations for AZD5305, a next-generation PARP1 selective inhibitor. Data for AZD5305 will demonstrate how the next wave of DNA damage response medicines can build on the success of PARP inhibitors, potentially allowing patients to stay on treatment longer.
” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Thousands of transcription factors — proteins that bind DNA and control gene expression — were studied in human cells. Molecular Therapy. Gene Therapy.
This removes the cancer’s blocking of the immuneresponse, so that T cells can fight the cancer. Keytruda’s classification based on mechanism, rather than its structure, is immune checkpoint inhibitor. Analyzing DNA patterns using AI may replace time-consuming process DNA and genomic sequencing tests, Yu said.
The end-of-year FDA approval of the first CRISPR-based therapy , for sickle cell disease, came a mere dozen years after Jennifer Doudna and Emmanuelle Charpentier introduced the technology. The microbes deploy them to dismantle the genetic material of infecting viruses, a little like an immuneresponse.
Everything started in school with an experiment on isolating DNA from bananas. Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences.
Coined immunotherapy, this umbrella term describes several types of treatment, including immune checkpoint inhibitors, immune system modulators, and adoptive cell therapies. Each triggers the immune system in a different way to fight cancer.
In the early 20th century, the discovery process was largely random , focused on cytotoxic agents that kill cells by disrupting key cellular functions like DNA replication. 5 This period also saw the rise of biologics in the 1980s, which have played a crucial role in modern therapies, particularly for diseases like cancer.
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.
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.
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.
The team at XinThera has developed research assets with the potential to target the DNA damage repair pathway in treating cancer and direct the body’s immuneresponse in inflammatory diseases, both of which may improve outcomes for people living with these diseases,” said Flavius Martin, M.D.,
.” Ming-Tain Lai, PhD, Chief Scientific Officer at OBI Pharma stated, “In the trial, OBI-833 demonstrated a favorable safety profile and generated detectable anti-Globo H IgM/IgG responses. ” Presentation number: 397P / Poster: ID 680. Additional information can be found at www.obipharma.com.
As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. Along with work on IFN-β protein, which became the multiple sclerosis drug Avonex ® , my team worked extensively on adenovirus-mediated IFN-β gene therapy.
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. The therapy is a combination of anticoagulant, anti-inflammatory and antiviral effects. FLT180a is an AAV gene therapy. They are also conducting Phase III trials.
Learning from Past Failures to Drive Toward a More Durable ImmuneResponse Following the initial clinical successes of blocking inhibitory receptors, like CTLA4 and PD1, many in the immuno-oncology field explored the potential to further enhance anti-tumor immunity by simultaneously enhancing co-stimulatory pathways.
NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided a year-end review and an outline of its plans for 2021. CARLSBAD, Calif.–( –( BUSINESS WIRE )– Lineage Cell Therapeutics, Inc.
(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 immuneresponses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.
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