MaxCyte, Inc.’s Post

Scientists have found a way to prevent the widespread loss of a targeted chromosome in human T cells due to CRISPR-Cas9 editing. ⬇️ - CRISPR-Cas9 technology allows researchers to edit genomic DNA at specific target sites, enabling the inactivation of genes, acquisition of new traits, and correction of genetic mutations. - One application of CRISPR-Cas9 is potentially boosting the immune response in cancer patients by engineering autologous T cells to target and destroy tumor cells. However, there are safety concerns regarding CRISPR-Cas9 genome editing, as improper repair of double-strand breaks (DSBs) can lead to the loss of targeted chromosomes. - In a study published in Cell, researchers found that Cas9-induced chromosomal loss was widespread when editing human T cells, with 5 to 20 percent of cells exhibiting partial or whole chromosome loss. Further investigation showed that chromosome loss occurred for almost 90 percent of all targets, regardless of the editing site, suggesting it is a general feature of Cas9 editing in T cells. ❗️The researchers investigated scRNA-seq data from a clinical trial and found no evidence of engineered T cell chromosome loss, which was surprising given previous findings, but they discovered that editing the cells before activation may have reduced chromosome loss. ✅ The researchers found that high TP53 expression after CRISPR-Cas9 editing correlated with lower chromosome loss rates, suggesting that modulating p53 during genome editing may protect against aneuploidy in CRISPR-engineered cells. https://lnkd.in/eQYTHJEs #cancer #cancerresearch #immunotherapy #immunooncology #crispr #genomeediting #cartcelltherapy #precisiononcology #sequencing

A new CRISPR gene-editing cell therapy may potentially cure Cancer, Sickle cell anemia, and other single-gene inheritance diseases. Using AI and High-Performance Computing to accelerate discovery, scientists can detect patterns in our genetic code, and work to pinpoint the switches that activate or block gene expression in our cells. Using CRISPR/Cas9 genetic scissors, scientists can then change DNA with extremely high precision. They can cut any DNA molecule at a predetermined site, then base edit the building blocks of our genetic code; altering the genetic instructions within our own body to cure disease. #tech4good #crispr #healthcare #innovation #cure #sicklecell #celltherapy #tcells, #basediting, #molecularbiology #curecancer #nobelprize #dna

Editing RNA instead of DNA signifies a major advance in gene-editing technology. By targeting transient messenger RNA, the CRISPR-based system not only enhances precision in cell therapies but also opens avenues for unraveling the intricate workings of gene interactions. Published in the recent Cell article, the results highlight a controlled and reversible impact, revitalizing exhausted CAR-T cells for therapeutic progress. #RNA #GeneEditing #CAR-T #CRISPR

This is so exciting and anyone who has taken UW Genome361 knows how much research and perseverance went into this. This is such an innovative and creative approach to circumnavigate the off target effects that have made SCA gene therapeutics so tough to bring to market. The bottom line? Bone marrow is extracted and stem cells undergo Cas9 edits to the BCL11A gene, which is active after fetal development to stop the production of HbF (fetus hemoglobin). Once deactivated and transplanted back into the patient, the edited cells engraft into the native bone marrow and increase the levels of HbF. Increased levels of HbF drastically decrease the sickling of red blood cells. This is done with a one time injection (after the most intense, straight-from-hell chemo on the market). Still, It is crazy to think that CRISPR has the power to reverse an early death sentence in a single session. I see a future where hereditary disorders don't come with a lifetime of medical trauma. I see a future where hereditary disorders have no effect on life at all. We are far from this point in a very multifaceted way, but this exciting approval definitely validates my hopes and dreams of Cas9 saving me from the cancer I know is waiting for me at 65. #crisprcas9 #drugdevelopment #geneediting #SCA

Big news! New research published today in Science Magazine highlights the potential of our systemic lung delivery and CFTR gene correction technology. This is the first study to show that lipid nanoparticles (LNPs) carrying gene editing tools can effectively correct a gene in lung stem cells. Daniel Siegwart, Ph.D., lead author and a co-founder of ReCode, led his team at UT Southwestern Medical Center, in collaboration with ReCode researchers, to develop SORT LNPs that could efficiently deliver gene-editing tools away from the liver and directly to cells in the lung. Their method showed over 70% editing efficiency in mice for correcting the genetic mutation (CFTR) that causes cystic fibrosis, which lasted nearly two years. We are now building on this data to translate SORT LNP-based gene editing into the clinical setting, leading to durable and potentially curative therapies for patients with genetic lung disorders. Learn more by reading the paper here:  https://lnkd.in/gxrTpGn4 Read our press release here: https://lnkd.in/gaQ56Bfk #CysticFibrosis #GeneticMedicine #GeneEditing

A new must read from Ned Pagliarulo and BioPharma Dive: Sickle cell disease is caused by genetic mutations that turn normally circular red blood cells into sharp-edged scythes that snag in vessels. In the most severe cases, these blockages can result in stroke, organ damage, and episodes of intense pain that recur again and again. Such pain episodes have been a lifelong peril for Elinam Joe Tsogbe, putting him in the hospital more than a dozen times some years. His disease led to a gallbladder removal at 14, a hip replacement at 19 and a dangerous trip to the intensive care unit in his early 30s. But over the past three years, Tsogbe hasn’t had any pain crises. In early 2021, he received an experimental transplant of his own stem cells, which had been collected and edited in a laboratory using CRISPR gene editing, biomedicine’s most cutting-edge tool. The treatment, called exa-cel and developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is a kind of genetic workaround for sickle cell, built on decades of research into the disease’s roots. Among the dozens of people who've received it in clinical testing, the effects are life-changing. Exa-cel is now up for FDA approval. It's clearance would be historic and usher in a new era of sickle cell treatment. Yet the therapy's benefits may not be accessible by all, or even most, of the tens of thousands of people estimated to have the disease in the U.S., never mind the millions more in other areas of the world where it is more prevalent. All this begs the question: What if a CRISPR cure isn’t such an easy choice? #biotech #medicine #geneediting https://lnkd.in/gvp5PB4S

In partnership with SelectScience, we recently spoke with Dr. Krishanu Saha, Associate Professor of Biomedical Engineering at the University of Wisconsin-Madison on how his team is using a nascent CRISPR-Cas9 gene editing method to generate CAR T-cells for the treatment of solid tumors. 🧬 Check out the full article to learn how this approach can circumvent the challenges of conventional viral-based methods and how tools from Thermo Fisher Scientific are helping advance Dr. Saha’s research toward clinical use.

If you've been following the landmark approval by regulators of the first #CRISPR gene therapy exa-cel by Vertex Pharmaceuticals and CRISPR Therapeutics to treat severe sickle cell disease (SCD), you also want to be aware of the following risks from the chemotherapy preconditioning with bisulfan that precedes treatment with exa-cel. No doubt a major milestone has been reached, but the work is far from complete. 1. risk of positive selection of mutant clones of hematopoietic stem cells (HSCs) that are linked to cancer 2. risk of lasting organ and tissue damage, including infertility Both of these risks are expected to increase with age. https://lnkd.in/e3zAerVK Link to the original journal article provided in the comments. 👇

#CRISPR gene editing has risks; here's a potentially safer alternative that changes #RNA instead of DNA. This might lead to more effective treatments (e.g. #cancer #treatment). https://lnkd.in/e2XsFm3e

Thoughts on this? >> IV infusion enables editing of the cystic fibrosis gene in lung stem cells >> Comment below! >>> lqventures.com #digitalhealth #socialmedia #digitalmarketing #industry40 #mhealth #IoT #healthtech #AI