#CRISPR-based therapies have countless applications for medical conditions, from rare genetic diseases and cancer to even common concerns like high cholesterol. “Great advances have been made toward enabling targeted knock-in of genes following CRISPR editing. Improved knock-in efficiency stems from advances in DNA template design along with optimization of delivery technologies and cell-handling protocols.” In a recent Biocompare article, James Brady, MaxCyte Senior Vice President, explains how CRISPR editing can improve gene knock-in efficiency, the critical players and activities necessary for successful editing, and the transfection of CRISPR reagents. Learn more: https://bit.ly/3VCxndY
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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
Avoiding Gene Editing’s Unintended Consequences
the-scientist.com
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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
FDA Approves First CRISPR Gene Editing Treatment for Sickle Cell Disease
scientificamerican.com
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Strategic and Tactical Leader | RNA, Cell & Gene Therapies | Business Development and E2E Drug Development & Manufacturing
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
MEGA-CRISPR tool gives a power boost to cancer-fighting cells
nature.com
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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
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
fda.gov
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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
In vivo editing of lung stem cells for durable gene correction in mice
science.org
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Targeted CRISPR activation is functional in engineered human pluripotent stem cells but undergoes silencing after differentiation into cardiomyocytes and endothelium These CRISPRa hiPSC lines effectively activate target genes in pluripotency, however the dCas9-VPR transgene expression is silenced after differentiation into cardiomyocytes and endothelial cells. To understand this silencing, we systematically tested different safe harbor sites and different promoters. Targeting to safe harbor sites hROSA26 and CLYBL loci also yielded hiPSCs that expressed dCas9-VPR in pluripotency but silenced during differentiation. Muscle-specific regulatory cassettes, derived from cardiac troponin T or muscle creatine kinase promoters, were also silent after differentiation when dCas9-VPR was introduced. In contrast, in cell lines where the dCas9-VPR sequence was replaced with cDNAs encoding fluorescent proteins, expression persisted during differentiation in all loci and with all promoters. Promoter DNA was hypermethylated in CRISPRa-engineered lines, and demethylation with 5-azacytidine enhanced dCas9-VPR gene expression. https://lnkd.in/esC8BKaW
Targeted CRISPR activation is functional in engineered human pluripotent stem cells but undergoes silencing after differentiation into cardiomyocytes and endothelium - Cellular and Molecular Life Sciences
link.springer.com
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#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
MEGA CRISPR: Engineering Better Immunotherapies with RNA Editing
the-scientist.com
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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
What if a CRISPR cure isn’t such an easy choice?
biopharmadive.com
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A milestone in genome editing has been achieved with the US debut of base editing in a clinical trial, which is testing complex genome edits to treat a difficult-to-treat form of leukemia. This groundbreaking technique offers precise and safer editing capabilities compared to traditional CRISPR–Cas9 methods, allowing researchers to make controlled changes to DNA sequences. While there are ongoing efforts to enhance base editing technology and address potential safety concerns, the field is rapidly evolving, with multiple groups exploring its therapeutic potential in treating various diseases, including cancer. This development marks a significant step forward in the world of gene editing and personalized medicine. #BaseEditing #CRISPR #ClinicalTrials #PrecisionMedicine #GeneTherapy #PersonalizedMedicine https://lnkd.in/edF5ua27
Super-precise CRISPR tool enters US clinical trials for the first time
nature.com
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Thoughts on this? >> IV infusion enables editing of the cystic fibrosis gene in lung stem cells >> Comment below! >>> lqventures.com #digitalhealth #socialmedia #digitalmarketing #healthtech #industry40 #IoT #AI #mhealth
IV infusion enables editing of the cystic fibrosis gene in lung stem cells
arstechnica.com
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