Biotech/Biomed Prediction #3
The PredictionMany disease-causing DNA changes (variants) within gene regulatory elements might be found to play a role in multiple tissues or organs simultaneously (i.e., pleiotropic effect) to drive a convergent disease phenotype, contrary to the belief that regulatory elements are highly tissue-specific. Example: Multiple variants control the expression of IRX3 and IRX5 genes in both human adipose tissue and brain, the coordinated effects of which (less thermogenesis and more feeding behav...
Biotech/Biomed Prediction #3
The PredictionMany disease-causing DNA changes (variants) within gene regulatory elements might be found to play a role in multiple tissues or organs simultaneously (i.e., pleiotropic effect) to drive a convergent disease phenotype, contrary to the belief that regulatory elements are highly tissue-specific. Example: Multiple variants control the expression of IRX3 and IRX5 genes in both human adipose tissue and brain, the coordinated effects of which (less thermogenesis and more feeding behav...
Biotech/Biomed Prediction #2
The PredictionIn the coming years, CRISPR will democratize therapeutic genome editing for n=1 genetic diseases. These efforts will be sponsored by academic institutions and nonprofits, as opposed to companies, because they won’t be profitable.Explainersn=1 genetic diseases: Ones that are so rare that there is likely only one, or up to a handful, cases diagnosed across the globe. Why democratizing genome editing is important: Since developing a treatment or cure for this kind of diseases only ...
Biotech/Biomed Prediction #2
The PredictionIn the coming years, CRISPR will democratize therapeutic genome editing for n=1 genetic diseases. These efforts will be sponsored by academic institutions and nonprofits, as opposed to companies, because they won’t be profitable.Explainersn=1 genetic diseases: Ones that are so rare that there is likely only one, or up to a handful, cases diagnosed across the globe. Why democratizing genome editing is important: Since developing a treatment or cure for this kind of diseases only ...
Biotech/Biomed Prediction #1
The PredictionIn 5-10 years, CRISPRa will treat or cure 10s to 100s of genetic diseases caused by haploinsufficiency in human patients. Example: SIM1 in extreme obesity. https://www.science.org/doi/10.1126/science.aau0629?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmedExplainersCRISPRa: CRISPRa, a technology derived from CRISPR, works like a light dimmer that can turn up the expression level of a given gene. Haploinsufficiency: Like matcha ice cream on a hot summer...
Biotech/Biomed Prediction #1
The PredictionIn 5-10 years, CRISPRa will treat or cure 10s to 100s of genetic diseases caused by haploinsufficiency in human patients. Example: SIM1 in extreme obesity. https://www.science.org/doi/10.1126/science.aau0629?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmedExplainersCRISPRa: CRISPRa, a technology derived from CRISPR, works like a light dimmer that can turn up the expression level of a given gene. Haploinsufficiency: Like matcha ice cream on a hot summer...
