BACKGROUND Primary intratracheal schwannoma is an extremely rare type of benign airway tumor,especially in adolescents.The presenting symptoms are typically prolonged cough and wheezing that can be misdiagnosed as ast...BACKGROUND Primary intratracheal schwannoma is an extremely rare type of benign airway tumor,especially in adolescents.The presenting symptoms are typically prolonged cough and wheezing that can be misdiagnosed as asthma in adolescent patients.CASE A 16-year-old adolescent girl admitted to a local hospital with symptoms of an irritating cough and wheezing was diagnosed with bronchial asthma and treated with budesonide and formoterol.Over the next year,the patient's wheezing and coughing symptoms gradually worsened and the antiasthma treatment was ineffective.One week prior to this admission,the patient developed dyspnea after catching a cold and was transferred to our hospital with a diagnosis of severe asthma.However,chest computed tomography and bronchoscopy showed a mass in the trachea.Primary intratracheal schwannoma was diagnosed by biopsy.Her symptoms were relieved by endoscopic resection by electrosurgical snaring combined with argon plasma coagulation.No relapse occurred during an 18 mo follow-up.CONCLUSION Primary intratracheal schwannoma should be considered in the differential diagnosis in adolescents with recurrent asthma-like attacks.展开更多
Small interfering RNA(siRNA)has demonstrated significant success in treating hepatic diseases through intravenous(i.v.)or subcutaneous(s.c.)injection.In this study,we reported that siRNA encapsulated in lipid nanopart...Small interfering RNA(siRNA)has demonstrated significant success in treating hepatic diseases through intravenous(i.v.)or subcutaneous(s.c.)injection.In this study,we reported that siRNA encapsulated in lipid nanoparticles(termed RBP131)can effectively achieve gene silencing in the liver via the non-invasive intratracheal(i.t.)administration.Following a single i.t.dose,apolipoprotein B(ApoB)gene silencing can be sustained for over three weeks,with a remarkably low effective dose(ED_(50))of 0.13 mg/kg.Multiple doses of 0.5 mg/kg administered twice a week can maintain longterm knockdown efficiency of around 90%.Further,in a hepatitis B virus(HBV)transgenic mouse model,significant X gene silencing(over 80%)was observed with the treatment of 1 mg/kg siHBVRBP131 formulation,comparable to the efficacy achieved through i.v.injection.Importantly,toxicity analysis indicated that the siRNA-RBP131 formulation was well tolerated in mice,even at a high dose of 10 mg/kg(over 70-times higher than ED_(50)).These findings open a new avenue for delivering siRNA-LNPs to the liver in a noninvasive strategy,providing a more patient-friendly approach that can be more easily translated into clinical practice.展开更多
基金Natural Science Foundation of Gansu Province,China,No.20JR5RA335.
文摘BACKGROUND Primary intratracheal schwannoma is an extremely rare type of benign airway tumor,especially in adolescents.The presenting symptoms are typically prolonged cough and wheezing that can be misdiagnosed as asthma in adolescent patients.CASE A 16-year-old adolescent girl admitted to a local hospital with symptoms of an irritating cough and wheezing was diagnosed with bronchial asthma and treated with budesonide and formoterol.Over the next year,the patient's wheezing and coughing symptoms gradually worsened and the antiasthma treatment was ineffective.One week prior to this admission,the patient developed dyspnea after catching a cold and was transferred to our hospital with a diagnosis of severe asthma.However,chest computed tomography and bronchoscopy showed a mass in the trachea.Primary intratracheal schwannoma was diagnosed by biopsy.Her symptoms were relieved by endoscopic resection by electrosurgical snaring combined with argon plasma coagulation.No relapse occurred during an 18 mo follow-up.CONCLUSION Primary intratracheal schwannoma should be considered in the differential diagnosis in adolescents with recurrent asthma-like attacks.
基金supported by the National Key Research and Development Program of China(No.2024YFB3814800)the National Natural Science Foundation of China(No.82272139)+2 种基金the“Open Competition to Select the Best Candidates”Key Technology Program for Cell and Gene Therapy of NCTIB(No.NCTIB2023XB02001)the Key Laboratory of Organ Regeneration and Reconstruction,Chinese Academy of Sciences(No.2024KF11)the Open Research Fund of Beijing Advanced Center of RNA Biology(BEACON)at Peking University.All schemes were created with BioRender.com.
文摘Small interfering RNA(siRNA)has demonstrated significant success in treating hepatic diseases through intravenous(i.v.)or subcutaneous(s.c.)injection.In this study,we reported that siRNA encapsulated in lipid nanoparticles(termed RBP131)can effectively achieve gene silencing in the liver via the non-invasive intratracheal(i.t.)administration.Following a single i.t.dose,apolipoprotein B(ApoB)gene silencing can be sustained for over three weeks,with a remarkably low effective dose(ED_(50))of 0.13 mg/kg.Multiple doses of 0.5 mg/kg administered twice a week can maintain longterm knockdown efficiency of around 90%.Further,in a hepatitis B virus(HBV)transgenic mouse model,significant X gene silencing(over 80%)was observed with the treatment of 1 mg/kg siHBVRBP131 formulation,comparable to the efficacy achieved through i.v.injection.Importantly,toxicity analysis indicated that the siRNA-RBP131 formulation was well tolerated in mice,even at a high dose of 10 mg/kg(over 70-times higher than ED_(50)).These findings open a new avenue for delivering siRNA-LNPs to the liver in a noninvasive strategy,providing a more patient-friendly approach that can be more easily translated into clinical practice.
