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Prime Assembly Expands Genome Editing Capabilities with Precise, Large-Scale DNA Integration

Prime Assembly Expands Genome Editing Capabilities with Precise, Large-Scale DNA Integration

A new genome engineering method, termed 'prime assembly,' has been developed to enable the precise and programmable integration of long DNA fragments into living cells. This advancement builds upon prime editing techniques, which typically allow for smaller insertions, deletions, and base swaps. Unlike many current genome editing methods that rely on untargeted gene delivery or short DNA edits, prime assembly utilizes CRISPR-targeted dual flap synthesis to write new DNA flaps into specific genomic locations. These flaps act as tethers, grabbing onto DNA fragments with matching ends, allowing for the precise insertion of one or more gene-sized DNA pieces. The method has been successfully applied to perform targeted exon recoding, transgene integration, and megabase-scale rearrangements in primary human cells, including at therapeutically relevant loci. A key advantage of prime assembly is its activity in both dividing and non-dividing cells, and its reduced likelihood of off-target effects compared to other...

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions to Accelerate Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions to Accelerate Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions to Accelerate Gene Editing Research

CRISPR-Based Gene Therapies Advance for Sickle Cell Disease and Beta-Thalassemia, Raising Ethical Questions on Germline Editing

CRISPR-Based Gene Therapies Advance for Sickle Cell Disease and Beta-Thalassemia, Raising Ethical Questions on Germline Editing

CRISPR-based gene therapies are increasingly being used in clinical settings for somatic conditions such as sickle cell disease and beta-thalassemia. The U.S. FDA approved Casgevy, the world's first CRISPR-based gene therapy, in December 2023 for sickle cell disease. This therapy involves editing somatic cells, meaning the modifications affect only the treated individual and are not heritable. However, the rapid advancement of gene-editing technology, particularly CRISPR-Cas9, has also brought germline editing closer to reality. Germline editing, which involves modifying sperm, eggs, or early-stage embryos, results in heritable changes that are passed down to future generations. This distinction is critical, as the person most affected by a germline edit does not yet exist at the time of the intervention, raising significant ethical and legal concerns regarding consent and long-term consequences. The controversial 2018 case of Chinese biophysicist He Jiankui, who edited the germline of human embryos, spark...

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions to Advance Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions to Advance Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions to Advance Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions for Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions for Gene Editing Research

Integrated DNA Technologies and Aldevron Launch New Cas9 mRNA Solutions for Gene Editing Research

CRISPR-Combo System Accelerates Gene-Edited Crop Regeneration, Addressing Agricultural Bottleneck

CRISPR-Combo System Accelerates Gene-Edited Crop Regeneration, Addressing Agricultural Bottleneck

Researchers from Texas A&M AgriLife Research, the University of Maryland, and the U.S. Department of Agriculture have developed a new system called CRISPR-Combo to significantly speed up the regeneration of gene-edited crops. Published in Nature Communications, this system addresses a major challenge in agricultural biotechnology: the slow and often unreliable process of growing a complete plant from gene-edited cells. CRISPR-Combo works by simultaneously editing a target gene and activating the plant's own morphogenic genes, which are responsible for cell division and development into roots, shoots, and full plants. Unlike previous methods that involved inserting additional growth-promoting genes or relying on external hormones, this new approach uses the existing CRISPR tool to enhance the activity of genes already present in the plant's genome. This method has shown promising results in various crops, including potato, citrus, strawberry, and poplar, leading to faster regeneration times and higher effic...

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

KAIST Research Uncovers Mechanism for RNA Delivery in Neurons, Impacting Brain Disorder Understanding

KAIST Research Uncovers Mechanism for RNA Delivery in Neurons, Impacting Brain Disorder Understanding

A research team at KAIST, led by Professor Ki-Jun Yoon, has identified a crucial mechanism by which neurons deliver RNA to distant regions. The study, published in Nature Communications, reveals that a small chemical modification on RNA, known as N6-methyladenosine (m6A), acts as a 'delivery tag.' This tag facilitates the transport of specific RNAs to distal axons, the long projections of neurons essential for transmitting signals. Unlike most other cells, neurons have extensive structures, requiring a precise system to ensure RNA reaches its correct destination. The team found that m6A, previously known to influence RNA stability and translation, also functions in this transport process. They observed that in mice where Mettl14, an enzyme component for m6A modification, was deleted, neurons exhibited impaired axon projection and neurite development, highlighting m6A's role in early neuronal development. The researchers further identified that the protein YTHDF2 recognizes m6A-modified RNA, and, in conjunc...

EPA Approves Gene-Edited Rootstock to Combat Citrus Greening in Florida

EPA Approves Gene-Edited Rootstock to Combat Citrus Greening in Florida

The Environmental Protection Agency (EPA) has approved a gene-edited rootstock designed to resist citrus greening bacteria, offering a potential solution for Florida's struggling citrus industry. Citrus greening, a bacterial infection spread by the Asian citrus psyllid, has devastated Florida's orange groves since its arrival in 2005, causing a drastic decline in fruit production. Yianni Lagos, from the company Soilcea in Tampa, Florida, developed the gene-edited rootstock, known as CarriCea T1, using CRISPR technology. This rootstock aims to maintain the tree's immune system, allowing it to fight off the greening bacteria and reduce reliance on insecticides. The focus on the rootstock is critical because the bacteria primarily damages the root system even before symptoms appear on the leaves. Growers are now planting trees with this gene-edited rootstock in test fields to observe its long-term performance.

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics is making significant strides in developing gene therapies for inherited retinal diseases (IRDs), specifically OPGx-LCA5 for LCA5-associated IRD and OPGx-BEST1 for BEST1-associated IRD. According to Dr. George Magrath, CEO of Opus Genetics, the OPGx-LCA5 program, targeting an ultra-rare disease affecting approximately 200 U.S. children, has shown promising results. All six participants treated to date (three pediatric and three adult) demonstrated improvement across various measures, including full-field stimulus threshold (FST) testing, best-corrected visual acuity (BCVA), virtual-reality maze tests, and microperimetry. Retinal sensitivity improved by about 1.5 log units at both blue and red wavelengths. The company has also secured FDA alignment for an eight-participant Phase 3 registrational trial for OPGx-LCA5, utilizing a 6-month pretreatment period where each participant serves as their own control. The OPGx-BEST1 program aims to restore bestrophin-1 channel function in the retinal pigm...

Preclinical Study Advances CRISPR-Cas9 Gene Editing for Wiskott-Aldrich Syndrome Towards Clinical Use

Preclinical Study Advances CRISPR-Cas9 Gene Editing for Wiskott-Aldrich Syndrome Towards Clinical Use

A comprehensive preclinical study has been published detailing the in-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing. This research, using Wiskott-Aldrich Syndrome as a model, established an integrated strategy for editing human hematopoietic stem cells (HSCs) and assessing genotoxicity. The study's findings have been instrumental in securing Orphan Drug Designation from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). This designation is a critical step in accelerating the development and review of therapies for rare diseases. The research focuses on the practical aspects of moving gene editing from laboratory settings to clinical application, emphasizing the need to demonstrate not only the effectiveness of the edit but also the potency, genotoxicity, and consistency of the process at a clinical scale. The project was driven by Asma Naseem, with significant contributions from collaborators including...