In a recent Cell article,McAuley,Kohn et al.showcased proof-of-principle of a promising gene therapy(GT)approach to correct the underlying genetic defect responsible for CD3δ-deficient severe combined immunodeficienc...In a recent Cell article,McAuley,Kohn et al.showcased proof-of-principle of a promising gene therapy(GT)approach to correct the underlying genetic defect responsible for CD3δ-deficient severe combined immunodeficiency(SCID)using a CRISPR-Cas9-derived adenine-base-editor(ABE).1 This ground-breaking and important study could impact future GTs for monogenic diseases by providing novel tailored therapeutic options(Fig.1).展开更多
A recent study published in Cell by the Kim lab represents a great advance towards expanding DNA editing possibilities to mitochondrial DNA(mtDNA)as they show reasonable targeted A-to-G conversions in human mtDNA usin...A recent study published in Cell by the Kim lab represents a great advance towards expanding DNA editing possibilities to mitochondrial DNA(mtDNA)as they show reasonable targeted A-to-G conversions in human mtDNA using base editors to broaden the arsenal of available tools.1 Mutations in mitochondrial DNA(mtDNA)are linked to several diseases(Fig.1),thus,development of gene therapy and editing approaches could lead to curative options for patients suffering from these illnesses.In this direction,gene editing tools such as zinc-finger nucleases(mitoZFN),transcription activator-like effector nucleases(mitoTALENs)and meganucleases(mitoARCUS)were shown to eliminate mutant mtDNA and shift heteroplasmy.展开更多
A recent study published in Cell by the Millay lab reports on an elegant strategy to expand the repertoire of lentiviral(LV)vectors from being the main delivery tool for ex vivo gene therapy to an in vivo applicable v...A recent study published in Cell by the Millay lab reports on an elegant strategy to expand the repertoire of lentiviral(LV)vectors from being the main delivery tool for ex vivo gene therapy to an in vivo applicable viral vector that specifically transduces skeletal muscle.1 This very significant advance may result in novel therapeutics for patients suffering from various skeletal muscle diseases.展开更多
Tumor metastases present an often difficult to treat therapeutic challenge for cancer patients and their caregivers.The recent paper by the Kaplan group(Kaczanowska et al.)exploited the natural capacity of monocytes o...Tumor metastases present an often difficult to treat therapeutic challenge for cancer patients and their caregivers.The recent paper by the Kaplan group(Kaczanowska et al.)exploited the natural capacity of monocytes or a subset of monocytes to migrate to tumor sites and pre-metastatic niches to deliver IL-12 in a novel anticancer approach designed to stimulate local immune responses.1 This raises the possibility to simultaneously fight cancer on at least two critical fronts,(1)elimination of the primary tumor and(2)inhibition of newly developing pre-metastatic lesions.展开更多
In a recent study published in Nature,Guan et al.1 showed an exciting strategy to reprogram human somatic cells into human chemically induced pluripotent stem cells(hCiPSCs).Thereby,they identified crucial steps and p...In a recent study published in Nature,Guan et al.1 showed an exciting strategy to reprogram human somatic cells into human chemically induced pluripotent stem cells(hCiPSCs).Thereby,they identified crucial steps and pathways that pose a barrier to chemically induced reprogramming of human cells.展开更多
文摘In a recent Cell article,McAuley,Kohn et al.showcased proof-of-principle of a promising gene therapy(GT)approach to correct the underlying genetic defect responsible for CD3δ-deficient severe combined immunodeficiency(SCID)using a CRISPR-Cas9-derived adenine-base-editor(ABE).1 This ground-breaking and important study could impact future GTs for monogenic diseases by providing novel tailored therapeutic options(Fig.1).
基金Open Access funding enabled and organized by Projekt DEAL.
文摘A recent study published in Cell by the Kim lab represents a great advance towards expanding DNA editing possibilities to mitochondrial DNA(mtDNA)as they show reasonable targeted A-to-G conversions in human mtDNA using base editors to broaden the arsenal of available tools.1 Mutations in mitochondrial DNA(mtDNA)are linked to several diseases(Fig.1),thus,development of gene therapy and editing approaches could lead to curative options for patients suffering from these illnesses.In this direction,gene editing tools such as zinc-finger nucleases(mitoZFN),transcription activator-like effector nucleases(mitoTALENs)and meganucleases(mitoARCUS)were shown to eliminate mutant mtDNA and shift heteroplasmy.
文摘A recent study published in Cell by the Millay lab reports on an elegant strategy to expand the repertoire of lentiviral(LV)vectors from being the main delivery tool for ex vivo gene therapy to an in vivo applicable viral vector that specifically transduces skeletal muscle.1 This very significant advance may result in novel therapeutics for patients suffering from various skeletal muscle diseases.
文摘Tumor metastases present an often difficult to treat therapeutic challenge for cancer patients and their caregivers.The recent paper by the Kaplan group(Kaczanowska et al.)exploited the natural capacity of monocytes or a subset of monocytes to migrate to tumor sites and pre-metastatic niches to deliver IL-12 in a novel anticancer approach designed to stimulate local immune responses.1 This raises the possibility to simultaneously fight cancer on at least two critical fronts,(1)elimination of the primary tumor and(2)inhibition of newly developing pre-metastatic lesions.
基金Open Access funding enabled and organized by Projekt DEAL.
文摘In a recent study published in Nature,Guan et al.1 showed an exciting strategy to reprogram human somatic cells into human chemically induced pluripotent stem cells(hCiPSCs).Thereby,they identified crucial steps and pathways that pose a barrier to chemically induced reprogramming of human cells.
