The field of advanced biotechnology,particularly genome editing,has witnessed transformative advancements in recent years^([1])with significant implications for the production of natural products^([2])and the enhancem...The field of advanced biotechnology,particularly genome editing,has witnessed transformative advancements in recent years^([1])with significant implications for the production of natural products^([2])and the enhancement of industrial biotechnology processes^([3]).To showcase these important works and inspire peers,we organized this special issue on Advances in Genome Editing Tools and Applications for Natural Products and Industrial Biotechnology.展开更多
Dear Editor,Genome editing holds potential to cure human diseases.The first CRISPR/Cas9 gene therapy,Casgevy,may offer a definitive treatment for hemoglobinopathies through autologous transplantation of ex vivo genome...Dear Editor,Genome editing holds potential to cure human diseases.The first CRISPR/Cas9 gene therapy,Casgevy,may offer a definitive treatment for hemoglobinopathies through autologous transplantation of ex vivo genome-corrected hematopoietic stem cells.However,in vivo editing of solid tissues has been marred by organ failures and deaths,poorly predicted by animal studies.1 The lack of reliable preclinical tools for human-specific therapies,such as genome editing,has remained a critical unmet need.Preclinical safety testing of genome editing could benefit from complex tissue models that bear the human genome and reconstitute human responses.Given the kidney’s susceptibility to toxic reactions from investigational drugs,studies using human stem cell-derived kidney organoids may inform clinical trials and improve patient outcome.2 Here,kidney organoids serve as a preclinical testing platform for CRISPR/Cas9 genome editing via adeno-associated virus(AAV)delivery,a common strategy in clinical trials that have raised safety concerns due to viral toxicity.展开更多
基金supported by grants from the National Key Research and Development Program of China(2021YFA0909500)the National Natural Science Foundation of China(32170080 and 32370026)TW would like to acknowledge funding by the Novo Nordisk Foundation(NNF20CC0035580).
文摘The field of advanced biotechnology,particularly genome editing,has witnessed transformative advancements in recent years^([1])with significant implications for the production of natural products^([2])and the enhancement of industrial biotechnology processes^([3]).To showcase these important works and inspire peers,we organized this special issue on Advances in Genome Editing Tools and Applications for Natural Products and Industrial Biotechnology.
文摘Dear Editor,Genome editing holds potential to cure human diseases.The first CRISPR/Cas9 gene therapy,Casgevy,may offer a definitive treatment for hemoglobinopathies through autologous transplantation of ex vivo genome-corrected hematopoietic stem cells.However,in vivo editing of solid tissues has been marred by organ failures and deaths,poorly predicted by animal studies.1 The lack of reliable preclinical tools for human-specific therapies,such as genome editing,has remained a critical unmet need.Preclinical safety testing of genome editing could benefit from complex tissue models that bear the human genome and reconstitute human responses.Given the kidney’s susceptibility to toxic reactions from investigational drugs,studies using human stem cell-derived kidney organoids may inform clinical trials and improve patient outcome.2 Here,kidney organoids serve as a preclinical testing platform for CRISPR/Cas9 genome editing via adeno-associated virus(AAV)delivery,a common strategy in clinical trials that have raised safety concerns due to viral toxicity.
