In vitro modeling of GM1 gangliosidosis using iPSC-derived cellular and organoid CNS models

Molecular Genetics and Metabolism(2023)

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摘要
Heart transplantation (HTx) outcomes have continuously improved to about 90%. HTx volumes have increased slowly, because of the mismatch between the number of candidate and donor organs. The use of temporary artificial life support (tALS) prior to HTx is increasing, with no difference in in-patient posttransplant mortality in the recent era between patients who did and did not receive tALS but with increased complications among those who received tALS. Wise application of new ALS technologies, both pre- and posttransplant, may improve the results, allowing a bridge to transplantation for unstable patients or a bridge to recovery and survival after severe early graft failure. ALS devices used before and after lung transplantation can be categorized as follows: implantable, paracorporeal devices used for long-term total support of the lungs; and intravenous devices used for a temporary or partial support. Generally, tools used as a bridge to lung transplantation are implantable paracorporeal devices, while intravenous devices are used for reversible lung diseases or an acute exacerbation of an existing chronic lung disease. Total support devices are more invasive than partial support devices.
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关键词
gm1 gangliosidosis,ipsc-derived
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