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A UCLA-led team has described how they advanced an experimental CRISPR approach for sickle cell disease into a blood stem cell therapy that can be manufactured at the scale and quality required for a clinical trial.
A UCLA-led team has described how it advanced an experimental CRISPR approach for sickle cell disease into a blood stem cell therapy that can be manufactured at the scale and quality required for a clinical trial.
Researchers built a cell-lined silicone blood vessel large enough to hold a real stent and quantified how endothelial cells recolonize the device under controlled blood-flow conditions.
Kamau Therapeutics, which has stayed relatively quiet since its 2023 emergence, is sharing clinical data from its revamped sickle cell gene editing program for the first time. The startup is giving a ...
Rice University's Han Xiao has used his AI-supported Sequence Display technology to expand the targeting abilities of CRISPR/Cas9. In a paper recently published in Nature Communications, Xiao evolved several variants of a compact Cas9 that could efficiently target stretches of DNA previously inaccessible for editing. The research included a proof-of-concept study in which the evolved Cas9s were successfully used to edit two genes found in coronary heart disease.
The action of a key gene is required to maintain a lifelong supply of stem cells that make bone healing possible, a new study finds.
Cryo-electron microscopy structures of the zebra finch R2 retrotransposon, alone and bound to Cas9, reveal how target-primed reverse transcription initiates and guided the engineering of an enhanced STITCHR gene-integration system.
Chick satellite cells from breast and leg muscles show opposite patterns of proliferation and differentiation when cultured under identical conditions, a new study finds.
We look at some of the key biotech developments, companies, and clinical data to watch at AHA Scientific Sessions 2026. The post From gene editing to RNA therapies: The biotech trends to look out for at AHA Scientific Sessions 2026 appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.
In this edition of AI Prognosis: These biotech startups don't think drug discovery is where AI can make the biggest difference.
A new review examines how plant-derived compounds such as polyphenols, flavonoids, alkaloids and isothiocyanates may target the breast cancer stem cells linked to recurrence, metastasis and treatment resistance.
A single gene-editing treatment could someday protect against heart disease, high cholesterol, and other common conditions for decades, potentially replacing lifelong medications. But scientists warn that permanently altering genes introduces risks that could take years to emerge, making long-term safety a critical challenge.
Scientists from Skoltech, together with colleagues from the Kharkevich Institute for Information Transmission Problems of the Russian Academy of Sciences, Lomonosov Moscow State University and other organizations, conducted a comparative study of the 3D genome organization in neurons derived from induced pluripotent stem cells and in neurons of the adult human brain. The authors collected and processed more than 300 3D genome maps covering 62 human samples and 34 mouse samples, including data on embryonic stem cells, induced pluripotent stem cells, neural progenitors, fetal and postmortem neurons, as well as neurons differentiated in vitro.
Researchers engineered lipid nanoparticles around the antihistamine hydroxyzine, achieving brain delivery of mRNA, CRISPR components and a radioprotective interleukin in animal models.
Researchers at the German Cancer Research Center (DKFZ) and the HI-STEM Stem Cell Institute have discovered a group of particularly long-lived blood stem cells in mice. These cells remain in a dormant state for an exceptionally long time while retaining their ability to generate new blood cells. This newly discovered cell population may be responsible for sustaining blood regeneration and immune defense throughout a mouse's entire life.
Some genetic diseases leave cells without a crucial protein. One possible treatment is to persuade genes already inside those cells to work harder, but the necessary machinery can be difficult to deliver, especially when researchers want to target several genes at once. A new compact CRISPR system devised by Hainan Zhang of the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, and colleagues fits those instructions into a single viral delivery package.
Shares of gene editing therapy companies mostly surged this past week on two growth catalysts: Data readouts at the European Society of Gene and Cell Therapy’s 33rd ESGCT Annual Congress, and the FDA's new draft guidance for personalized gene therapies. The post StockWatch: Gene Editing Shares Jump on ESGCT Data, FDA Draft Guidance appeared first on GEN - Genetic Engineering and Biotechnology News.
In the third of a four-part Health Matters wellness series on frontier medicine in Hong Kong, Fiona Sun examines how a local start-up is testing a gene-editing therapy that could tackle a group of inherited eye diseases at their genetic source and what it will take to turn it into a treatment patients can access. For 15 years, Professor Zheng Zongli has worked to make gene-editing therapies safe enough to treat diseases with limited treatment options. His path has taken him from a PhD in medical...
Archaea Behind Complex Life The microbes closest to the ancestor of complex life turn out to be plain
Liu ensured that his return visit to ESGCT (having spoken at the congress in Barcelona in 2019) would be one of the highlights of the conference. The “meta lesson” of the past few years in therapeutic genome editing is that different scientific approaches and applications can synergize with regulatory, manufacturing, and economic advances “to achieve the mission we’re on.” The post No Nonsense: David Liu Lauds Progress in Disease-Agnostic Gene Editing at ESGCT appeared first on GEN - Genetic Engineering and Biotechnology News.
We are pleased to announce that Dr. Johnson V. John, Assistant Professor at the Terasaki Institute for Biomedical Innovation (TIBI), has received an R56 grant from the National Eye Institute (NEI) of the National Institutes of Health (NIH).
Cells are very particular about the proper locations of their contents. For example, DNA is packaged in the nucleus in a highly specific way, but researchers are still trying to understand how this happens. For stem cells in the brain of a larval fruit fly—a model organism used to study processes such as early development—the DNA is organized so that the center of each strand, called the centromere, is at the top of the nucleus.
By: Victoria Tenor In January 2026, Canada took a step forward in agricultural biotechnology by approving the country’s first gene-edited (GE) mammal for human consumption. … The post Canada Approves its First Gene-edited Pig for Human Consumption appeared first on SAIFood.
A new review maps how mutations in more than twenty genes weaken bone in osteogenesis imperfecta and surveys therapies from bisphosphonates to CRISPR gene editing.
A new study shows that the RNA-binding protein MSI1 stabilizes the ferritin-like transcript Fthl17c, preserving the ferrous iron supply that TET enzymes need to keep DNA hypomethylated and mouse embryonic stem cells pluripotent.
A preclinical CRISPR screen found that loss of mTOR-regulating genes sensitizes small cell lung cancer cells to PARP inhibitors, and combining the autophagy inhibitor GNS561 with AZD5305 slowed tumors and improved survival in mouse models.
The FDA's draft guidance could streamline approval of gene-editing therapies tailored to individual mutations, while families and experts weigh safety, affordability, evidence requirements, and access challenges.
The first patient received a dose of the high-precision gene editor in a new clinical trial for Wilson disease. Researchers hope it's a one-and-done fix. The post CRISPR Prime Editing Rewrites DNA in the Human Body for the First Time appeared first on SingularityHub.
A planetary-scale mining of public metagenomes collected 794 million CRISPR spacers and 1.18 billion virus and plasmid hits, revealing high spacer turnover and evidence that some targeting reflects viral genomes entering non-host cells.
In rat bone marrow stem cells, the TLR4 inhibitor TAK-242 suppressed LPS-induced inflammatory gene expression while leaving osteogenic differentiation largely unimpaired by day 14.
Scientists can now edit genes in precise locations deep in the brain and body without the use of invasive surgery or implants, thanks to new work from researchers at the Wu Tsai Neurosciences Institute. The team demonstrated its technique, which uses a combination of ultrasound, light-emitting nanoparticles and photo-controllable gene-editing proteins, by successfully altering genes in mouse brains at selected locations.
A new review summarizes how basal stem cells sustain lifelong olfactory epithelium renewal and how single-cell and organoid technologies are reshaping the field.
A survey of 1,218 consumers in Spain, Germany and the UK found that conventional pesticides draw stronger rejection than precision-bred watermelon, with zero-residue pesticides commanding the highest premiums.
A new review examines how patient-derived tumor organoids retaining immune cells could improve preclinical prediction of immunotherapy response while noting major standardization and validation hurdles.
Researchers fine-mapped and functionally validated CauYL, a magnesium chelatase H subunit gene essential for chlorophyll accumulation and normal leaf coloration in cauliflower.
Researchers generated eyeless F0 CRISPR-Cas9 pax6 mutants in the slipper limpet Crepidula fornicata using a de novo transcriptome instead of an assembled genome.
The Biomolecular Nanotechnology research group led by Ikerbasque research professor Aitziber L. Cortajarena has developed a new iron-based contrast agent for magnetic resonance imaging that can be used for both diagnosis and treatment. In a study carried out on lab-grown human heart tissue, it was proven that the contrast agent accumulates in the fibrotic tissue, that its signal intensity on the MRI image increases with the severity of the disease, and that it slows disease progression.
Researchers built a human blood-brain barrier model combined with antiviral testing, validated it with anti-HIV drugs, showed TBEV crosses an intact barrier to infect astrocytes, and identified a barrier-permeable antiviral candidate.
CRISPR Prime Editing: First In Vivo Trial for Wilson’s Disease For years, scientists have been working on ways to treat genetic disorders that cause serious health problems. Now, a Phase 1/2 clinical trial is testing an experimental prime-editing treatment called PM577a. So far, only one patient has received the treatment, and the first clinical results […] The post CRISPR Breakthrough: First In Vivo Prime Editing Treatment Tested in Humans appeared first on BioTecNika.
A one-hour exposure to activated protein C keeps human blood stem cells quiescent and improves their short-term serial engraftment in mice, while thrombin acts on the same receptor to drive differentiation and impair transplantation outcomes.
A new review in Biological Research surveys cardiac stem and progenitor cell populations, finding that classic markers c-kit and Sca-1 mostly label endothelial cells without cardiomyogenic potential.
A new review maps how 3D bone marrow models, from spheroids to bioprinted and organ-on-chip systems, are being used to study multiple myeloma and its drug resistance.
A new closed-tube RT-RAA-CRISPR/Cas12a assay detects Schmallenberg virus within 50 minutes and outperformed RT-qPCR on simulated bovine nasal swab samples, though validation with authentic clinical specimens is still pending.
Japanese researchers have established the first patient-derived organoid model of canine nasal carcinoma, showing variable drug sensitivities among tumor lines and identifying S100A2 as a promoter of cancer cell proliferation and invasion.
A dynamic bicellular in vitro model linked by a fluidic circuit reproduced vascular disruption and fibroblast changes when endothelial cells were stimulated with immune complexes from systemic sclerosis patients.
A cell-culture study reports that prodigiosin reduced apoptosis and oxidative stress in BMSCs exposed to chemically induced hypoxia, with effects linked to HIF-1α/YAP-associated signaling.
Researchers report generating human chemically induced pluripotent stem cells from umbilical cord-derived mesenchymal stem cells using an optimized small-molecule protocol under normal oxygen conditions.
A new review finds CRISPR engineering can sharply raise carotenoid yields in microalgae, yet these gains mostly redistribute carbon rather than increase net CO2 fixation.
Scientists used CRISPR/Cas9 to disrupt the tyr and oca2 pigmentation genes in zebrafish, reducing melanophores and visibly altering coloration in F0 and F1 mutants.
Researchers report that inhibiting the methyltransferase PRMT5 with EPZ015666 keeps mouse, monkey and human spermatogonia proliferating in an undifferentiated state in vitro, with the effect reversible on drug withdrawal.
A new review argues that neural stem cells may aid spinal cord injury recovery mainly by reprogramming macrophages and other immune cells rather than replacing lost neurons.
A new study of 14 Microcoleus species finds that CRISPR–Cas systems are widespread but do not follow species phylogeny, pointing to repeated gain, loss and exchange of components through gene flow.
A mouse study found that hematopoietic stem cells engrafted during active bone marrow regeneration showed superior long-term reconstitution compared with cells engrafted after myeloablative conditioning.
A new study maps about 1,100 sites where PRC2 rapidly re-establishes itself in mouse embryonic stem cells after reversible depletion of the core subunit Suz12.
Engineered three-dimensional muscle bundles grown from pediatric patients with juvenile dermatomyositis, immune-mediated necrotizing myopathy, and arthritis-related weakness show measurable donor-specific functional and structural differences in a proof-of-concept study.
A genomic survey of thirteen complete Kluyvera genomes found that only Kluyvera ascorbata strain SK carries a complete type I-E CRISPR-Cas locus, whose spacers mostly match plasmid sequences from related enteric bacteria.
An engineered compact Cas12m variant enables multiplexed CRISPR gene activation from a single AAV vector in mouse models.
Braided electrospun PU/ALMA scaffolds carrying decellularized tendon ECM and umbilical cord stem cells showed stronger mechanics, slower degradation and better tendon regeneration in rabbits.
A new MCDA-CRISPR/Cas12a assay detects the aminoglycoside resistance gene armA in about 70 minutes with 100 percent concordance on 129 sequenced clinical isolates.
The FDA has outlined steps drugmakers can take to seek regulatory approval for gene editing medicines tailored to fix a single person's disease-causing mutation. The announcement is part of an ongoing ...
CDMOs already play a critical role in the cell and gene therapy development and manufacturing space and, says Rose Biosolutions, with emerging bioprocessing technologies, production methods, and strategies, are creating even more opportunities for contractors. The post Decentralization, AI, and Gene Editing are Opportunities for CGT Contractors appeared first on GEN - Genetic Engineering and Biotechnology News.
A Hiroshima University researcher outlines advantages of Japan's new law that prohibits the transfer of genome-edited human embryos or germ cells into the human reproductive tract and imposes criminal penalties for violations.
A Hiroshima University researcher contends that Japan's newly enacted law regulating human genome-edited embryos and germ cells marks a significant advance in the governance of reproductive genome editing. Tsutomu Sawai, professor at Hiroshima University's Graduate School of Humanities and Social Sciences, praises the new law for defining prohibited activities and penalties for violation but also stresses the need for stronger international coordination to prevent the prohibitions from being circumvented by individuals seeking procedures abroad.
Four seriously ill Chinese boys who would almost certainly have died in their twenties from a rare inherited disease received the same highly anticipated gene-editing drug. More than a year later, a set of cruel and contradictory data emerged. Two boys gained the strength to walk farther. One boy showed no improvement in gait and subsequently withdrew from the trial. And the fourth boy’s heart stopped beating 81 hours after receiving the medication intravenously, owing to a severe systemic...
Interim Phase 3 results from 80 women suggest Humacyte's lab-grown blood vessel provided usable dialysis access sooner than fistulas, reducing catheter time, though independent review is still pending.
A gene-edited Monascus strain fermented for 21 days showed the strongest glucose uptake and lipid improvements in insulin-resistant liver cells, though the mechanisms remain candidate associations.
A genome-wide CRISPR screen identifies UGCG-driven sphingolipid metabolism and protective autophagy as drivers of Niraparib resistance in ovarian cancer.
Two LAMP-CRISPR diagnostic pipelines for Mycobacterium tuberculosis reached a limit of detection of 10 copies per microlitre, with the Cas12a version outperforming the Cas13a pipeline on clinical samples.
A study in BMC Medical Genomics finds that miR-183-5p is upregulated in the serum of adolescent idiopathic scoliosis patients, shows diagnostic potential, and inhibits bone-forming differentiation of human bone marrow mesenchymal stem cells by negatively regulating GNG12.
University of Missouri researchers used the AI-designed, open-source gene editor OpenCRISPR-1 to create rice plants that resist bacterial blight, with commercial varieties still years away.
The AI company Anthropic has acquired a drug discovery startup called Mithril Medicine, as part of its push into the life sciences. And that's where things start to get strange. Mithril is ...
University of Missouri researchers have created rice plants that resist bacterial blight, a devastating disease that threatens one of the world's most important food crops. The advance could help farmers in parts of Asia and Africa, where the disease can destroy harvests and threaten livelihoods.
A new review maps how sensor-embedded 3D-bioprinted tracheal grafts, supported by AI and digital twins, could transform airway reconstruction while warning that the technology remains experimental.
Discover our top picks of biotech incubators in Europe giving life science startups the boost they need to get off the ground. The post 13 of the best biotech incubators hatching startups in Europe appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.
A pH- and ROS-responsive hydrogel carrying CRISPR-Cas9 silenced the TRPV4 calcium channel in rat discs, restoring autophagic flux and slowing degeneration over eight weeks.
A three-dimensional co-culture of human blastoids and endometrial assembloids reveals reciprocal transcriptional adaptations, including SDC1-positive MMP7-positive trophoblast subsets linked to stromal CSF3 signalling, at the onset of implantation-like interaction.
A new review in the Journal of Translational Medicine examines how cancer stem cells, vascularization and immune evasion contribute to therapeutic resistance in central nervous system tumors.
A new review maps four molecular routes by which CRISPR systems can detect cancer-linked DNA methylation in cell-free DNA, and finds that none has yet reached independent clinical validation.
A randomised phase 1 clinical trial conducted by scientists and doctors from Shanghai and Guangzhou has achieved encouraging results in the treatment of Type 1 diabetes using stem cells extracted from the mouth’s gum tissue. More than half of those who received the treatment showed varying degrees of improvement, while one subject was able to discontinue exogenous insulin therapy completely, the researchers said. Results of the study, which tested a therapy based on gingival mesenchymal stem...
A new doctoral dissertation by Omar Anwar Elkadi in the field of Applied Physics on near-infrared spectroscopy for the continuous monitoring of tissue-engineered cartilage will be defended at the Faculty of Science, Forestry and Technology, Kuopio campus, of the University of Eastern Finland on Oct. 30.
Scientists edited the promoter of the rice gene OsGTL1 to lower stomatal density, raising instantaneous water use efficiency while revealing a trade-off between water conservation and yield components.
Human Wharton's jelly-derived mesenchymal stem cells delivered inside an alginate-gelatin hydrogel improved follicular architecture and lowered inflammatory cytokines in a rat model of polycystic ovary syndrome, a preliminary animal study reports.
New findings raise concerns about the potential effects of maternal COVID-19 infection on early nervous system development.
Recovery and transplantation of organs following vitrification using cryoprotectant is an important step along the road to the future technological capability of whole body vitrification and recovery - and recovery of those individuals already preserved at low temperature by the cryonics industry. It is particularly important because it is a highly desirable capability for the organ transplant industry, and thus a way to bring significant funding and credibility to the endeavor of cryonics, the preservation of people for future revival. Scaling up vitrification and rewarming to human sized organs has been a major challenge on several fronts, such as effective perfusion of cryoprotectant in large tissues, performance of the cryoprotectant, consistent warming of deeper tissue in a large vitrified mass, and so forth. It is […]
A review integrates murine, human, and bovine evidence on epithelial and mesenchymal stem cells in the mammary gland, highlighting mTORC1-dependent metabolic regulation, niche interactions, and experimental therapeutic approaches for bovine mastitis.
Warhound says gene editing and cloning can fix why military dog candidates fail, but questions about genes and oversight remain.
How can the identity of pluripotent stem cells be determined more reliably during their differentiation? Researchers from the IUF—Leibniz Research Institute for Environmental Medicine and Heinrich Heine University Duesseldorf have systematically validated marker genes for this purpose and developed a web-based application for the automated classification of early cell fates. The study has been published in Stem Cell Reports.
A systematic bibliometric analysis of 25 years of Ficus literature shows that only about 11.3 percent of publications address genomic or genetic engineering topics and that CRISPR-related keywords are absent from the field's thematic maps, revealing a translational bottleneck between rapidly expanding genomic resources and functional genome editing in figs.
Human cells hold about two meters (6.6 feet) of DNA, folded and compacted to fit inside a nucleus. That packing is essential for life, but it creates a problem for scientists. Some of the genes that control a cell's identity are buried so deep in tightly packed DNA that gene-targeting tools such as CRISPR activation, or CRISPRa, struggle to reach them.
Warhound came out of stealth on Wednesday with $200 million in funding and a mission to create "elite working dogs" equipped with "advanced body armor."
Huntington's disease is caused by repetitive "stutters" in DNA—specifically repeated CAG sequences—that expand abnormally over time, leading to toxic protein accumulation that contributes to the progression of the disease. There is currently no cure for this rare and fatal genetic disease.
Human neural stem cells that help form the brain and spinal cord are highly susceptible to SARS-CoV-2, including omicron variants BA.1 and BA.2, according to new research from the University of California, Riverside. The findings raise concerns about the potential effects of maternal COVID-19 infection on early nervous system development and underscore the importance of monitoring children exposed to the virus before birth.
A controlled study of 70 male rats found that stem cells led early wound contraction, while chitosan achieved the strongest contraction and marker results by day 14.
A three-layer bioprinted conduit containing living Schwann cells restored sciatic nerve function in rats over 16 weeks with results close to those of a natural nerve autograft.
A structured review found that CRISPR diagnostics for maternal–infant HIV, hepatitis B and syphilis care remain clinically unready, with HIV early infant diagnosis offering the strongest near-term development opportunity.
Gene editing is moving from lab bench to approved therapies. But how should sponsors measure editing efficiency without unnecessary complexity? The post Great gene editing. Now prove it without overcomplicating the analysis appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.
Experiments in mouse-derived astrocytes and human embryonic stem cells identify GLIS3 as an upstream regulator of astrocyte differentiation and neural gene-expression pathways.
Gene therapy is no longer the future of medicine—it's reality. Treatments that replace, silence or edit genes are already changing clinical care.
A 2026 review summarizes advances in CRISPR-Cas9 precision, delivery and primary-cell engineering for genetic diseases and diabetes, while the available abstract does not provide quantitative results or detailed study methods.