Researchers at UMass Chan Medical School have developed a microRNA-based gene therapy that suppresses mutant SOD1 production, delaying disease onset by 60 days and extending lifespan by 100 days in mice models of ALS. The therapy, delivered via adeno-associated virus (AAV) vector, preserves motor neurons and maintains neuromuscular con...
SourceUMass Chan Medical School·JournalNature Communications·TypeExperimental study·DateJul 29, 2026
Emerging non-endocytic delivery strategies enable direct cytosolic delivery of proteins, nucleic acids, and gene-editing tools, providing new opportunities for biomedical therapies. The review discusses their application prospects in gene therapy, macromolecular drug delivery, and cellular engineering.
SourceBiomedical Analysis·JournalBiomedical Analysis·TypeLiterature review·DateJul 24, 2026
Researchers at Mass General Brigham have been awarded $25.8 million in ARPA-H funding to develop a scalable gene editing platform for treating rare genetic vascular diseases. The VESSEL program aims to create durable, potentially one-time treatments for severe genetic vasculopathies.
Researchers have successfully produced genome-edited T cells that can target and destroy cancer cells using the Platinum TALEN genetic engineering technique. The technique was found to be efficient, with an average yield of 72 million 1G4-TCR cells for every 3 million T-cells.
SourceHiroshima University·JournalCytotherapy·TypeExperimental study·DateJul 6, 2026
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The 2026 Warren Alpert Foundation Prize was awarded to five scientists who developed curative genetic therapies for two major blood disorders. These treatments, CASGEVY and LYFGENIA, have been approved by the FDA for use in the US and have shown significant advancements in the field of gene therapy.
Researchers developed a novel fluorescent-based method to analyze upstream open reading frames (uORFs) in plant genomes, enabling rapid analysis of gene regulation. The technique simplifies current methods by using intact leaf tissue and fluorescent proteins, reducing sample preparation and consumable materials.
SourceCarl R. Woese Institute for Genomic Biology, University of Illinois at Urbana-Champaign·JournalPlant Direct·TypeExperimental study·DateJun 22, 2026
Researchers developed a gene therapy that restored normal brain activity and improved behavior in mice with Fragile X syndrome by replacing the missing FMRP protein. The treatment administered during early development showed significant improvements in cognitive flexibility, social interactions, and probabilistic reversal learning.
SourceUniversity of California - Riverside·JournalMolecular Therapy — Nucleic Acids·TypeExperimental study·DateJun 18, 2026
The University of Virginia has joined SPARK GLOBAL to leverage resources and expertise in accelerating the development of new medicines. This collaboration aims to reduce time from lab discoveries to clinic trials, delivering tangible healthcare solutions to patients.
Researchers have discovered that plant viruses can deliver CRISPR RNA guides to plants, enabling targeted genome edits. The study showcases the potential of potyvirus-based systems for expanding crop genome editing.
SourceNanjing Agricultural University The Academy of Science·JournalHorticulture Research·DateJun 14, 2026
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Researchers identified a promising new strategy for reversing autism-related brain deficits by targeting a specific glycine transporter. The therapy restored NMDA receptor function in mouse models and human brain organoids, improving behavioral abnormalities such as social interaction and repetitive behaviors.
SourceInstitute for Basic Science·JournalNature Communications·TypeExperimental study·DateJun 9, 2026
The SMArT platform achieves near-pure selection of correctly edited blood stem cells while reducing dangerous genomic alterations. The innovative strategy enables enrichment of cells with targeted integration to 100% purity.
SourceFondazione Telethon·JournalNature Biotechnology·TypeExperimental study·DateJun 1, 2026
Researchers successfully created a novel, stably inheritable grass carp germplasm without intermuscular bones using the runx2b gene. The absence of IBs did not affect the fish's quality or nutritional profile, but improved gel strength and resilience were observed.
SourceScience China Press·JournalScience China Life Sciences·TypeExperimental study·DateMay 27, 2026
Philip Tai, PhD, received a $1.6 million grant to investigate AAV vector mechanisms using high-resolution DNA sequencing technology. His findings could lead to new vector designs that improve gene therapy treatments' safety. The goal is to remove mutations that cause cancer-causing integration into host cells.
A research team at the University of Zurich has developed a new gene editing approach that correctly treats the genetic mutation causing hereditary epilepsy in mice. The therapy improves communication between nerve cells, reduces febrile seizures, and increases survival rates.
SourceUniversity of Zurich·JournalScience Translational Medicine·TypeExperimental study·DateMay 13, 2026
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Researchers have developed a new gene editing technology called 'prime assembly' that allows efficient insertion of large DNA segments into the human genome. This innovation enables treatment of genetic diseases by replacing entire genes, promising substantial progress in treating conditions with multiple mutations.
SourceUMass Chan Medical School·JournalNature·TypeExperimental study·DateMay 13, 2026
A preclinical study using precision gene editing targets and corrects the root cause of Dravet syndrome, a rare form of childhood epilepsy. The approach demonstrates significant success in treating the disease, including improved survival rates and reduced seizures.
SourceJackson Laboratory·JournalScience Translational Medicine·TypeExperimental study·DateMay 13, 2026
Toloo Taghian, a UMass Chan Medical School assistant professor, has received a five-year, $3.2 million grant to develop a gene therapy for UBA5 disorder, a rare genetic disease that affects protein balance and leads to neurological impacts. The goal is to monitor disease progression and determine the efficacy of future clinical trials.
Researchers summarize universal mechanisms of regulatory T cells in solid organ transplantation, enabling durable immune tolerance and reducing rejection. Gene-editing technologies create hypoimmunogenic Tregs for standardized, scalable availability.
SourceImmunity & Inflammation·JournalImmunity & Inflammation·TypeSystematic review·DateMay 8, 2026
A new CRISPR protein, Cas12a2, has shown potential for killing sick cells while leaving healthy ones untouched. Researchers have tested its effectiveness in destroying cancer cells and virus-infected cells with promising results.
SourceUniversity of Utah Health·JournalNature·TypeExperimental study·DateMay 6, 2026
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Researchers at Gladstone Institutes identified hundreds of human genes influencing HIV infection and two potent antiviral proteins, PI16 and PPID. These proteins block HIV's entry into T cells or limit its ability to replicate within the cell.
Researchers have made a breakthrough in developing a novel platform that harnesses the immune system's ability to produce therapeutic proteins. By editing hematopoietic stem cells with CRISPR gene-editing tools, they were able to create a long-term, boostable source of antibodies capable of protecting against deadly influenza infection...
SourceRockefeller University·JournalScience·DateApr 16, 2026
Scientists corrected a genetic disease of the liver in mouse models and human patient cells using gene editing, building a foundation for a potential new therapy for Zellweger spectrum disorder. The breakthrough restored function of the liver and peroxisomes, reducing toxic buildup.
SourceJackson Laboratory·JournalNature Biomedical Engineering·TypeExperimental study·DateApr 15, 2026
Researchers have discovered a smaller CRISPR enzyme, Al3Cas12f, that can efficiently edit genes in human cells. The enzyme's unique structure allows it to form a stable connection with DNA, making it a promising candidate for therapeutic genome editing.
SourceUniversity of Texas at Austin·JournalNature Structural & Molecular Biology·TypeExperimental study·DateApr 13, 2026
Researchers used base editing to correct the SCN8A gene mutation responsible for severe inherited epilepsy. The approach successfully eliminated or reduced seizures and improved brain function in lab mice, offering new hope for treating genetic epilepsies.
SourceUniversity of Virginia Health System·JournalJournal of Clinical Investigation·DateApr 7, 2026
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A gene-edited treatment has shown remarkable success against severe sickle cell disease, with 27 out of 28 patients achieving a functional cure and no painful crises. The therapy uses CRISPR/Cas12a technology to modify stem cells and increase levels of fetal hemoglobin.
SourceCleveland Clinic·JournalNew England Journal of Medicine·DateApr 1, 2026
Researchers at University of Michigan Engineering and Michigan Medicine used protein nanoparticles to genetically modify several types of human cells, including liver cancer and immune cells. The goal is to develop a safer method for delivering gene therapies without using modified viruses.
SourceUniversity of Michigan·JournalAdvanced Materials·DateMar 30, 2026
Researchers at USC will develop an AI-driven framework to strengthen evidence generation for gene and cell therapies, helping bring promising treatments closer to patients. The project aims to better understand how specific therapy features relate to patient outcomes.
Researchers have discovered an RNA-guided CRISPR system that can activate genes without cutting DNA, opening up new possibilities for gene regulation and therapeutic strategies. The system uses a strand of RNA as a guide to recruit the cell's transcription machinery, allowing for precise control over gene expression.
A new approach, called INSTALL, enables non-toxic DNA integration in multiple human cell types and successfully inserts large genetic payloads in mice, offering a promising solution for genetic therapies. The study's findings have the potential to broaden the applicability of genome editing therapies.
SourceMass General Brigham·JournalNature·TypeExperimental study·DateMar 11, 2026
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Genethon's GNT0004 gene therapy shows long-term efficacy in patients with Duchenne muscular dystrophy, maintaining clinical efficacy and safety at two years. The trial included 72 boys aged 6-10 with retained walking ability, treated with GNT0004 at a therapeutic dose.
The company's ATA-200 gene therapy has shown safety, pharmacodynamics, and efficacy results in the first patients treated, offering hope for children with LGMD-R5. The therapy delivers a normal copy of the γ-sarcoglycan gene and has been awarded Orphan Drug Designation in the US and Europe.
A new rice gene, GSN7, has been discovered that can simultaneously increase grain yield and improve quality. The study found that precise manipulation of this gene can overcome the long-standing trade-off between yield and quality in rice breeding.
SourceScience China Press·JournalScience Bulletin·DateFeb 23, 2026
Engineers have refined a technology to edit individual genetic base pairs, reducing unintended edits and increasing safety for potential treatments. The new base editors could lead to better outcomes for some cystic fibrosis patients and more accurate models for drug testing.
SourceUniversity of Pennsylvania School of Engineering and Applied Science·JournalMolecular Therapy·TypeExperimental study·DateFeb 23, 2026
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Nikon Monarch 5 8x42 Binoculars deliver bright, sharp views for wildlife surveys, eclipse chases, and quick star-field scans at dark sites.
UCLA researchers have developed a novel gene-editing approach using lipid nanoparticles to deliver a full-length CFTR gene into human airway cells. The study shows promise for treating cystic fibrosis by correcting the underlying genetic mutation, which could lead to more effective and long-term therapies.
SourceUniversity of California - Los Angeles Health Sciences·JournalAdvanced Functional Materials·DateFeb 17, 2026
David J. Segal has been appointed as the chair of UC Davis Department of Biochemistry and Molecular Medicine, known for his groundbreaking research in gene-editing technologies. He is developing targeted molecular tools to treat rare genetic disorders, including Angelman syndrome and neurofibromatosis type 1.
SourceUniversity of California - Davis Health·DateFeb 5, 2026
Researchers developed a gene-editing therapy that directly corrects genetic mutations responsible for autosomal dominant polycystic kidney disease, slowing cyst growth and improving health outcomes. The therapy uses CRISPR-based base editing to precisely correct single-letter DNA mutations in the PKD1 gene.
SourceMayo Clinic·JournalNature Communications·DateJan 29, 2026
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Researchers at the University of British Columbia have developed a topical CRISPR-based therapy that can correct faulty genes in human skin, potentially treating genetic skin conditions like ARCI and eczema. The treatment, using lipid nanoparticle technology, restores up to 30% of normal skin function.
SourceUniversity of British Columbia·JournalCell Stem Cell·TypeExperimental study·DateJan 27, 2026
The ISSCR is hosting a summit on access and affordability in cell and gene therapies to explore pricing, manufacturing, regulation, and reimbursement strategies. Experts will examine key barriers and emerging solutions across the access landscape.
SourceInternational Society for Stem Cell Research·DateJan 27, 2026
Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells. Genethon's gene therapy approach has shown preclinical efficacy in animal studies, correcting glycogen accumulation and improving cardiac hypertrophy and muscular dysfunction.
Researchers at St. Jude Children's Research Hospital created CHANGE-seq-BE to evaluate the activity and specificity of base editors, a genome editing technology, ensuring safety and accuracy. The technique outperformed conventional approaches and has already been used in clinical work.
SourceSt. Jude Children's Research Hospital·JournalNature Biotechnology·TypeExperimental study·DateJan 2, 2026
A new treatment using genome-edited immune cells has shown promising results in helping children and adults fight a rare form of blood cancer called T-cell acute lymphoblastic leukaemia (T-ALL). The gene therapy, known as BE-CAR7, uses base-editing to modify T-cells and destroy cancerous cells.
SourceUniversity College London·JournalNew England Journal of Medicine·DateDec 8, 2025
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Researchers have made significant advances in genetically modified plants that produce alpha-amylase inhibitor proteins, making them indigestible to pests like bedbugs, beetles, weevils, and woodworms. Gene editing techniques, such as CRISPR, offer a promising solution to combat insect pests without compromising human consumption.
SourceFundação de Amparo à Pesquisa do Estado de São Paulo·JournalBiotechnology Journal·DateNov 12, 2025
Plant biologists developed a method to grow transgenic plants in weeks instead of months by hijacking a plant's natural regeneration abilities. The technique uses bacteria carrying genetic instructions for wound healing and regeneration to trigger plant growth.
SourceCell Press·JournalMolecular Plant·TypeExperimental study·DateNov 6, 2025
Researchers at the University of Texas at Austin have developed a novel gene-editing method that can correct multiple disease-causing mutations simultaneously. This approach uses bacterial retrons to protect the microbes from viral infection and has shown promising results in correcting scoliosis-causing mutations in zebrafish embryos.
SourceUniversity of Texas at Austin·JournalNature Biotechnology·TypeExperimental study·DateOct 23, 2025
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Researchers create enhanced T cells with improved survival in cancer models by simultaneously modifying multiple genes using CRISPRoff and CRISPRon. The approach overcomes toxicity issues associated with traditional gene editing methods, enabling high cell survival rates and potential for treating various diseases.
SourceArc Institute·JournalNature Biotechnology·TypeExperimental study·DateOct 21, 2025
Researchers at MIT have developed a new system that allows for precise control over the expression of synthetic genes in cells. The DIAL system uses a promoter editing mechanism to establish desired protein levels, which can be edited after delivery. This technology has the potential to improve gene therapy and cell reprogramming appli...
SourceMassachusetts Institute of Technology·JournalNature Biotechnology·DateOct 14, 2025
A pioneering case of pig-to-human liver xenotransplantation has been successfully demonstrated, with the genetically engineered porcine liver functioning for an extended period in a human recipient. The patient survived for 171 days despite complications such as xenotransplantation-associated thrombotic microangiopathy.
SourceElsevier·JournalJournal of Hepatology·TypeCase study·DateOct 9, 2025
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Researchers at CNIO have created a 'human repairome', a catalogue of 20,000 DNA 'scars' that reveal how genes affect DNA repair. This information can help determine the best treatment for each cancer type and overcome resistance to therapy.
SourceCentro Nacional de Investigaciones Oncológicas (CNIO)·JournalScience·TypeExperimental study·DateOct 2, 2025
Mass General Brigham has identified key areas of innovation in medicine, including gene editing, immune system modulation, AI-powered care, and organ transplantation. The 'Big Ideas in Medicine' aim to transform diagnosis, treatment, and prevention of disease with next-generation technologies.
A UCLA research team led by Dr. Donald Kohn has developed a one-time stem cell gene therapy treatment for alpha thalassemia major that could be curative. The therapy involves adding the missing alpha-globin gene to patient cells using a viral vector, enabling them to produce functional hemoglobin.
SourceUniversity of California - Los Angeles·JournalCell Reports Medicine·TypeExperimental study·DateSep 17, 2025
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Aranet4 Home CO2 Monitor tracks ventilation quality in labs, classrooms, and conference rooms with long battery life and clear e-ink readouts.
Researchers found that mutations in the CFAP410 gene change its interaction with another protein, making motor neuron cells more vulnerable to DNA damage and cell death. This discovery provides new insights into the mechanisms underlying Motor Neurone Disease and highlights potential targets for new therapies.
SourceUniversity of Bath·JournaliScience·TypeExperimental study·DateSep 8, 2025
Researchers at Northwestern University have developed a new CRISPR delivery system that triples efficiency using DNA-wrapped nanoparticles, improving safety and effectiveness. The new system, called LNP-SNAs, targets specific cells and tissues, reducing toxicity and boosting gene-editing efficiency by threefold.
SourceNorthwestern University·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateSep 1, 2025
Researchers at EPFL developed BindCraft, an open-source AI platform that uses AlphaFold2 to generate novel binders with desired functional properties. The platform reduces the need for high-throughput screening and makes protein design more democratized.
SourceEcole Polytechnique Fédérale de Lausanne·JournalNature·DateAug 29, 2025
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Rigol DP832 Triple-Output Bench Power Supply powers sensors, microcontrollers, and test circuits with programmable rails and stable outputs.
Researchers have successfully treated damage caused by heart attacks in non-human primates using gene therapy, restoring both strength and rhythm of the damaged hearts. The treatment improved heart function in pathological conditions with no adverse effects observed.
SourceDuke University·JournalCirculation Research·TypeExperimental study·DateAug 28, 2025
A study published in the Journal of Hepatology reveals that only 15-20% of neonatal liver cells are responsible for generating over 90% of the adult liver mass. This finding has major implications for pediatric gene therapy, allowing scientists to achieve more effective and durable correction of inherited liver diseases.
SourceFondazione Telethon·JournalJournal of Hepatology·TypeExperimental study·DateAug 28, 2025
Researchers at U. Iowa identify a specific metabolic pathway that fuels tumor growth in MPNST, providing a promising new target for treatment. By blocking this pathway, tumors grow more slowly and are more vulnerable to chemotherapy.
SourceUniversity of Iowa Health Care·JournalScience Advances·TypeExperimental study·DateAug 18, 2025
Researchers developed a one-time gene editing treatment that restored hearing and balance in adult mice with DFNA41, a genetic form of progressive deafness. The therapy successfully disabled the harmful mutation while preserving the normal gene, leading to long-term hearing and balance restoration.
SourceMass Eye and Ear·JournalJournal of Clinical Investigation·TypeExperimental study·DateAug 14, 2025
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Researchers developed a CRISPR-based gene-editing system that changes a single molecule within mosquitoes, halting malaria-parasite transmission. The new system is designed to genetically spread the malaria resistance trait until entire populations of the insects no longer transfer the disease-causing parasites.
SourceUniversity of California - San Diego·JournalNature·TypeExperimental study·DateJul 23, 2025
A team of scientists proposes an integrated framework combining biotechnology and AI to revolutionize crop breeding, exploring multi-omics, genome editing, and high-throughput phenotyping. The authors present a forward-looking framework for AI-assisted crop germplasm design, offering a roadmap for sustainable agriculture.
SourceChinese Academy of Sciences Headquarters·JournalNature·TypeLiterature review·DateJul 23, 2025
A recent study identified 37 human-specific genes linked to brain development and cognitive abilities, which are hijacked by cancer to drive tumor growth. The researchers discovered nearly half of these genes become aberrantly activated in cancerous tissues, promoting tumor cell proliferation.
SourceChinese Academy of Sciences Headquarters·JournalCell Genomics·TypeExperimental study·DateJul 18, 2025