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Rockefeller University Develops Stem Cell Editing for Long-Term Therapeutic Protein Production

Rockefeller University Develops Stem Cell Editing for Long-Term Therapeutic Protein Production

Rockefeller University Develops Stem Cell Editing for Long-Term Therapeutic Protein Production
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ThermoCas9 Gene Editing Technology Advances with Methylation-Sensitive Tools

ThermoCas9 Gene Editing Technology Advances with Methylation-Sensitive Tools

ThermoCas9 Gene Editing Technology Advances with Methylation-Sensitive Tools
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New CRISPR Tool Developed to Target Tumor DNA with Precision

New CRISPR Tool Developed to Target Tumor DNA with Precision

A team of scientists from Wageningen University & Research and Van Andel Institute has developed a CRISPR variant called ThermoCas9, which selectively targets and cuts tumor DNA while sparing healthy DNA. This method exploits cancer-specific DNA methylation patterns, marking an early but promising step toward high-precision cancer therapies. The study, published in Nature, indicates the potential for future advancements in cancer treatment. ThermoCas9 distinguishes between unmethylated and methylated genes, allowing it to target tumor cells specifically. The research demonstrated that ThermoCas9 could cut DNA in tumor cells while leaving healthy DNA intact, showcasing its ability to detect subtle chemical differences between healthy and tumor cells.
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Rockefeller University Researchers Develop Platform to Map Genetic Variations in Cancer

Rockefeller University Researchers Develop Platform to Map Genetic Variations in Cancer

Researchers at The Rockefeller University have developed a platform called PerturbFate, which systematically maps how genetic variations associated with diseases like melanoma reshape cells. This study, published in Nature, focuses on identifying regulatory nodes common to diverse genetic variations, offering a path toward combination therapies that can target diseases across multiple genetic causes. The platform allows researchers to observe genetic changes in real-time by tracking DNA accessibility and RNA production in single cells. By focusing on melanoma drug resistance, the team identified 143 genes linked to resistance to the drug Vemurafenib. The study found that targeting common control points significantly reduced drug resistance, suggesting a promising strategy for combination therapies.
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CRISPR Variant ThermoCas9 Targets Tumor DNA, Offering Precision in Cancer Therapy

CRISPR Variant ThermoCas9 Targets Tumor DNA, Offering Precision in Cancer Therapy

A team of researchers from Van Andel Institute and Wageningen University & Research has developed a CRISPR variant known as ThermoCas9, which can distinguish between tumor DNA and healthy DNA, selectively cutting only the former. This advancement, published in Nature, represents a significant step towards high-precision cancer therapies. The method exploits the differences in DNA methylation, a process where small chemical tags regulate gene activity, which is altered in cancer cells. ThermoCas9, discovered by Dr. John van der Oost, can be programmed to locate and cut specific DNA sections, effectively targeting tumor cells while sparing healthy ones. This is the first CRISPR-based method to use methylation for targeting human cancer cells.
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Rockefeller University Researchers Develop Platform to Target Melanoma Drug Resistance

Rockefeller University Researchers Develop Platform to Target Melanoma Drug Resistance

Researchers at The Rockefeller University have developed a platform called PerturbFate, which systematically maps how genetic variations associated with diseases reshape cells. The study, published in Nature, focuses on melanoma drug resistance, identifying regulatory nodes common to diverse genetic variations. This approach offers a path toward combination therapies that can target diseases across various genetic causes. The platform allows researchers to observe genetic changes in real-time, tracking DNA accessibility and RNA production. By analyzing over 300,000 cells, the study found that diverse genetic perturbations pushed melanoma cells into a drug-resistant state, which could be mitigated by targeting common control points.
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CRISPR Variant ThermoCas9 Targets Tumor DNA, Promising Precision Cancer Therapy

CRISPR Variant ThermoCas9 Targets Tumor DNA, Promising Precision Cancer Therapy

Researchers from Wageningen University & Research and Van Andel Institute have developed a novel gene-editing approach using a CRISPR variant called ThermoCas9. This method exploits the chemical differences between tumor DNA and healthy DNA, specifically targeting and cleaving tumor DNA while sparing normal tissue. The key to this precision lies in DNA methylation, where methyl groups attach to DNA, regulating gene activity. In cancer cells, these methylation patterns become aberrant, providing a molecular fingerprint for targeting. ThermoCas9, discovered in bacteria, is sensitive to these methylation patterns, allowing it to distinguish between healthy and malignant DNA. The research demonstrated ThermoCas9's ability to selectively cleave tumor DNA in cultured human cells, marking a significant advance in precision medicine.
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Rockefeller University Unveils PerturbFate Platform for Cancer Drug Resistance

Rockefeller University Unveils PerturbFate Platform for Cancer Drug Resistance

Researchers at The Rockefeller University have developed PerturbFate, a platform that maps genetic variations in cells to identify common regulatory nodes. This technology tracks gene regulation in single cells over time, revealing shared control points that can be targeted for combination therapies. The study, focusing on melanoma drug resistance, demonstrated that targeting these nodes significantly reduces drug resistance. The platform also highlighted the role of the Mediator Complex in drug resistance, offering new insights into potential therapeutic strategies.
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Neion Bio's Innovative Approach to Biologics Manufacturing Using Chickens

Neion Bio's Innovative Approach to Biologics Manufacturing Using Chickens

Neion Bio's Innovative Approach to Biologics Manufacturing Using Chickens
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University of Texas Researchers Develop Miniature CRISPR for Enhanced Gene Editing in Human Cells

University of Texas Researchers Develop Miniature CRISPR for Enhanced Gene Editing in Human Cells

Researchers at the University of Texas at Austin have engineered a compact version of the CRISPR-Cas12f nuclease, which shows promise for efficient gene editing in human cells. This development addresses a significant challenge in gene therapy: the large size of traditional CRISPR nucleases, which limits their delivery via adeno-associated virus (AAV) vectors. The newly developed Cas12f variant, named Al3Cas12f, is approximately one-third the size of the commonly used Cas9 enzyme. Despite its smaller size, Al3Cas12f demonstrated robust activity, achieving over 50% editing efficiency at numerous genomic sites and exceeding 90% at several targets. The research, published in Nature Structural & Molecular Biology, highlights the potential of this miniature enzyme to overcome current limitations in in vivo gene therapy applications.
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ProImmune and University of Texas Medical Branch Collaborate to Enhance Infectious Disease Research

ProImmune and University of Texas Medical Branch Collaborate to Enhance Infectious Disease Research

ProImmune, Ltd., a leader in immunological reagents, has announced a collaboration with The University of Texas Medical Branch (UTMB) Galveston National Laboratory (GNL) to advance research on high-consequence infectious diseases. This partnership will focus on the development and evaluation of Ankyrons, a novel class of binding reagents, as molecular tools for precise detection and study of viral proteins. The collaboration aims to enhance understanding of viral protein function and immune responses, which are crucial for developing next-generation vaccines and therapeutics. The research will initially target pathogens of global health concern, including various ebolaviruses and the Mpox virus.
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CRISPR Gene Editing Trials Show Promise in Treating Cardiovascular Diseases

CRISPR Gene Editing Trials Show Promise in Treating Cardiovascular Diseases

Recent trials involving CRISPR gene editing have demonstrated significant potential in treating various cardiovascular diseases. A single dose of mRNA-encoded CRISPR therapy, known as nexiguran ziclumeran, has shown rapid and durable reductions in serum transthyretin (TTR) levels, which is promising for the treatment of ATTR cardiomyopathy (ATTR-CM). Additionally, CRISPR-Cas9 in vivo gene editing has been effective in treating transthyretin amyloidosis. Another study highlighted the use of mRNA-encoded CRISPR base editing of PCSK9, which efficiently and durably reduced LDL cholesterol levels in non-human primates. These advancements underscore the potential of CRISPR technology in revolutionizing gene therapy for cardiovascular diseases.