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UID:23169-1659096000-1659099600@scienceinvancouver.com
SUMMARY:Development & Utilization of In Vivo Systems to Optimize Lipid Nanoparticles for Therapeutic Genome Editing
DESCRIPTION:Genetic diseases are a leading cause of death and disability in Canada with immense economic and societal burdens. Gene therapy has emerged as a means to effectively treat genetic diseases; however\, current gene therapies are limited by their high manufacturing costs\, the inability to re-dose\, and the safety concerns of some viral vectors. \nCRISPR genome editing is a new therapeutic approach that aims to directly repair the underlying disease-causing mutations. Conventional CRISPR methods are limited as in vivo therapeutics because they introduce DNA breaks and cause frequent off-target edits. Newer base editors and prime editors overcome the limitations traditional CRISPR genome editing methods because they do not introduce DNA breaks. \nHowever\, the delivery of genome editors to affected tissues remains a challenge. Viral vectors\, such as AAV\, are unsuitable for genome editing because their long expression (years) increases the probability of unintended edits. In contrast\, the transient expression (hours-days) of RNA encoding genome editors via nanoparticles is well suited for genome editing\, and unlike viral vectors\, nanoparticles can be readministered. However\, nanoparticle delivery of complex genome editing cargos (large mRNA + small gRNA) remains a challenge\, especially to extra-hepatic target tissues such as muscle. To address this\, we are developing new ways to safely deliver these new editors using lipid nanoparticles. \nTo efficiently measure the in vivo effectiveness of genome editor delivery via LNPs\, we have developed transgenic mice that carry mutations in reporter genes. Precise gene repair of these mutations produce a functional enzyme that emits light (luminescence) that sensitive imagers can detect to precisely measure the location and extent of gene editing in living animals. We have made progress in our goal towards efficient and safe in vivo genome editing that we will share. \n\n\n\n\n\n\nDr. Colin Ross seeks to incorporate genomics into guiding\, optimising\, and developing novel therapeutics to improve the safety and effectiveness of medications. His research is also exploring the use of genome sequencing to help diagnose and manage the treatment of rare genetic diseases.
URL:https://scienceinvancouver.com/event/development-utilization-of-in-vivo-systems-to-optimize-lipid-nanoparticles-for-therapeutic-genome-editing/
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CREATED:20220713T164118Z
LAST-MODIFIED:20220713T164118Z
UID:26574-1659096000-1659099600@scienceinvancouver.com
SUMMARY:Development & Utilization of In Vivo Systems to Optimize Lipid Nanoparticles for Therapeutic Genome Editing
DESCRIPTION:Genetic diseases are a leading cause of death and disability in Canada with immense economic and societal burdens. Gene therapy has emerged as a means to effectively treat genetic diseases; however\, current gene therapies are limited by their high manufacturing costs\, the inability to re-dose\, and the safety concerns of some viral vectors. \nCRISPR genome editing is a new therapeutic approach that aims to directly repair the underlying disease-causing mutations. Conventional CRISPR methods are limited as in vivo therapeutics because they introduce DNA breaks and cause frequent off-target edits. Newer base editors and prime editors overcome the limitations traditional CRISPR genome editing methods because they do not introduce DNA breaks. \nHowever\, the delivery of genome editors to affected tissues remains a challenge. Viral vectors\, such as AAV\, are unsuitable for genome editing because their long expression (years) increases the probability of unintended edits. In contrast\, the transient expression (hours-days) of RNA encoding genome editors via nanoparticles is well suited for genome editing\, and unlike viral vectors\, nanoparticles can be readministered. However\, nanoparticle delivery of complex genome editing cargos (large mRNA + small gRNA) remains a challenge\, especially to extra-hepatic target tissues such as muscle. To address this\, we are developing new ways to safely deliver these new editors using lipid nanoparticles. \nTo efficiently measure the in vivo effectiveness of genome editor delivery via LNPs\, we have developed transgenic mice that carry mutations in reporter genes. Precise gene repair of these mutations produce a functional enzyme that emits light (luminescence) that sensitive imagers can detect to precisely measure the location and extent of gene editing in living animals. We have made progress in our goal towards efficient and safe in vivo genome editing that we will share. \n\n\n\n\n\n\nDr. Colin Ross seeks to incorporate genomics into guiding\, optimising\, and developing novel therapeutics to improve the safety and effectiveness of medications. His research is also exploring the use of genome sequencing to help diagnose and manage the treatment of rare genetic diseases.
URL:https://scienceinvancouver.com/event/development-utilization-of-in-vivo-systems-to-optimize-lipid-nanoparticles-for-therapeutic-genome-editing-2/
LOCATION:Online
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DTSTART;TZID=America/Vancouver:20220729T120000
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DTSTAMP:20260615T202449
CREATED:20220713T164118Z
LAST-MODIFIED:20220713T164118Z
UID:28189-1659096000-1659099600@scienceinvancouver.com
SUMMARY:Development & Utilization of In Vivo Systems to Optimize Lipid Nanoparticles for Therapeutic Genome Editing
DESCRIPTION:Genetic diseases are a leading cause of death and disability in Canada with immense economic and societal burdens. Gene therapy has emerged as a means to effectively treat genetic diseases; however\, current gene therapies are limited by their high manufacturing costs\, the inability to re-dose\, and the safety concerns of some viral vectors. \nCRISPR genome editing is a new therapeutic approach that aims to directly repair the underlying disease-causing mutations. Conventional CRISPR methods are limited as in vivo therapeutics because they introduce DNA breaks and cause frequent off-target edits. Newer base editors and prime editors overcome the limitations traditional CRISPR genome editing methods because they do not introduce DNA breaks. \nHowever\, the delivery of genome editors to affected tissues remains a challenge. Viral vectors\, such as AAV\, are unsuitable for genome editing because their long expression (years) increases the probability of unintended edits. In contrast\, the transient expression (hours-days) of RNA encoding genome editors via nanoparticles is well suited for genome editing\, and unlike viral vectors\, nanoparticles can be readministered. However\, nanoparticle delivery of complex genome editing cargos (large mRNA + small gRNA) remains a challenge\, especially to extra-hepatic target tissues such as muscle. To address this\, we are developing new ways to safely deliver these new editors using lipid nanoparticles. \nTo efficiently measure the in vivo effectiveness of genome editor delivery via LNPs\, we have developed transgenic mice that carry mutations in reporter genes. Precise gene repair of these mutations produce a functional enzyme that emits light (luminescence) that sensitive imagers can detect to precisely measure the location and extent of gene editing in living animals. We have made progress in our goal towards efficient and safe in vivo genome editing that we will share. \n\n\n\n\n\n\nDr. Colin Ross seeks to incorporate genomics into guiding\, optimising\, and developing novel therapeutics to improve the safety and effectiveness of medications. His research is also exploring the use of genome sequencing to help diagnose and manage the treatment of rare genetic diseases.
URL:https://scienceinvancouver.com/event/development-utilization-of-in-vivo-systems-to-optimize-lipid-nanoparticles-for-therapeutic-genome-editing-3/
LOCATION:Online
END:VEVENT
BEGIN:VEVENT
DTSTART;TZID=America/Vancouver:20220729T120000
DTEND;TZID=America/Vancouver:20220729T130000
DTSTAMP:20260615T202449
CREATED:20220713T164118Z
LAST-MODIFIED:20220713T164118Z
UID:31272-1659096000-1659099600@scienceinvancouver.com
SUMMARY:Development & Utilization of In Vivo Systems to Optimize Lipid Nanoparticles for Therapeutic Genome Editing
DESCRIPTION:Genetic diseases are a leading cause of death and disability in Canada with immense economic and societal burdens. Gene therapy has emerged as a means to effectively treat genetic diseases; however\, current gene therapies are limited by their high manufacturing costs\, the inability to re-dose\, and the safety concerns of some viral vectors. \nCRISPR genome editing is a new therapeutic approach that aims to directly repair the underlying disease-causing mutations. Conventional CRISPR methods are limited as in vivo therapeutics because they introduce DNA breaks and cause frequent off-target edits. Newer base editors and prime editors overcome the limitations traditional CRISPR genome editing methods because they do not introduce DNA breaks. \nHowever\, the delivery of genome editors to affected tissues remains a challenge. Viral vectors\, such as AAV\, are unsuitable for genome editing because their long expression (years) increases the probability of unintended edits. In contrast\, the transient expression (hours-days) of RNA encoding genome editors via nanoparticles is well suited for genome editing\, and unlike viral vectors\, nanoparticles can be readministered. However\, nanoparticle delivery of complex genome editing cargos (large mRNA + small gRNA) remains a challenge\, especially to extra-hepatic target tissues such as muscle. To address this\, we are developing new ways to safely deliver these new editors using lipid nanoparticles. \nTo efficiently measure the in vivo effectiveness of genome editor delivery via LNPs\, we have developed transgenic mice that carry mutations in reporter genes. Precise gene repair of these mutations produce a functional enzyme that emits light (luminescence) that sensitive imagers can detect to precisely measure the location and extent of gene editing in living animals. We have made progress in our goal towards efficient and safe in vivo genome editing that we will share. \n\n\n\n\n\n\nDr. Colin Ross seeks to incorporate genomics into guiding\, optimising\, and developing novel therapeutics to improve the safety and effectiveness of medications. His research is also exploring the use of genome sequencing to help diagnose and manage the treatment of rare genetic diseases.
URL:https://scienceinvancouver.com/event/development-utilization-of-in-vivo-systems-to-optimize-lipid-nanoparticles-for-therapeutic-genome-editing-4/
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DTSTAMP:20260615T202449
CREATED:20220713T164118Z
LAST-MODIFIED:20220713T164118Z
UID:32732-1659096000-1659099600@scienceinvancouver.com
SUMMARY:Development & Utilization of In Vivo Systems to Optimize Lipid Nanoparticles for Therapeutic Genome Editing
DESCRIPTION:Genetic diseases are a leading cause of death and disability in Canada with immense economic and societal burdens. Gene therapy has emerged as a means to effectively treat genetic diseases; however\, current gene therapies are limited by their high manufacturing costs\, the inability to re-dose\, and the safety concerns of some viral vectors. \nCRISPR genome editing is a new therapeutic approach that aims to directly repair the underlying disease-causing mutations. Conventional CRISPR methods are limited as in vivo therapeutics because they introduce DNA breaks and cause frequent off-target edits. Newer base editors and prime editors overcome the limitations traditional CRISPR genome editing methods because they do not introduce DNA breaks. \nHowever\, the delivery of genome editors to affected tissues remains a challenge. Viral vectors\, such as AAV\, are unsuitable for genome editing because their long expression (years) increases the probability of unintended edits. In contrast\, the transient expression (hours-days) of RNA encoding genome editors via nanoparticles is well suited for genome editing\, and unlike viral vectors\, nanoparticles can be readministered. However\, nanoparticle delivery of complex genome editing cargos (large mRNA + small gRNA) remains a challenge\, especially to extra-hepatic target tissues such as muscle. To address this\, we are developing new ways to safely deliver these new editors using lipid nanoparticles. \nTo efficiently measure the in vivo effectiveness of genome editor delivery via LNPs\, we have developed transgenic mice that carry mutations in reporter genes. Precise gene repair of these mutations produce a functional enzyme that emits light (luminescence) that sensitive imagers can detect to precisely measure the location and extent of gene editing in living animals. We have made progress in our goal towards efficient and safe in vivo genome editing that we will share. \n\n\n\n\n\n\nDr. Colin Ross seeks to incorporate genomics into guiding\, optimising\, and developing novel therapeutics to improve the safety and effectiveness of medications. His research is also exploring the use of genome sequencing to help diagnose and manage the treatment of rare genetic diseases.
URL:https://scienceinvancouver.com/event/development-utilization-of-in-vivo-systems-to-optimize-lipid-nanoparticles-for-therapeutic-genome-editing-5/
LOCATION:Online
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