Biologics : Targets & Therapy最新文献

筛选
英文 中文
Successful Response to Golimumab in a Case of Relapsing Polychondritis Overlapping with Ulcerative Colitis. 复发性多软骨炎与溃疡性结肠炎重叠病例对戈利木单抗的成功应答
IF 4
Biologics : Targets & Therapy Pub Date : 2024-01-12 eCollection Date: 2024-01-01 DOI: 10.2147/BTT.S436301
Shu Kojima, Satohiro Matsumoto, Yudai Koito, Takaya Miura, Masanari Sekine, Takeshi Uehara, Takeharu Asano, Yasuhiro Yamaguchi, Hirosato Mashima
{"title":"Successful Response to Golimumab in a Case of Relapsing Polychondritis Overlapping with Ulcerative Colitis.","authors":"Shu Kojima, Satohiro Matsumoto, Yudai Koito, Takaya Miura, Masanari Sekine, Takeshi Uehara, Takeharu Asano, Yasuhiro Yamaguchi, Hirosato Mashima","doi":"10.2147/BTT.S436301","DOIUrl":"10.2147/BTT.S436301","url":null,"abstract":"<p><p>A 51-year-old Japanese man was diagnosed with left-sided ulcerative colitis (UC) at age 41. He was treated with mesalazine and azathioprine and maintained remission. At age 51, the patient developed bloody stools, abdominal pain, scleritis, arthritis, cough, bloody sputum, and pericardial effusion. Considering that pericardial effusion is an atypical extraintestinal complication of UC, and the patient met the diagnostic criteria for relapsing polychondritis (RP), a diagnosis of RP complicating a relapse of UC was made. Steroid therapy was administered, and both diseases improved. Golimumab, an anti-tumor necrosis factor-α inhibitor, was introduced as maintenance therapy for UC. All symptoms, including pericardial effusion, improved. Subsequently, no relapse of UC or RP was observed. As only a few cases of RP overlapping with UC have been reported and no treatment protocol has been established, we considered this case valuable and worthy of publication.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10790666/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139485113","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Hypoxia Inducible Factor-1α Through ROS/NLRP3 Pathway Regulates the Mechanism of Acute Ischemic Stroke Microglia Scorching Mechanism. 缺氧诱导因子-1α通过ROS/NLRP3途径调控急性缺血性卒中小胶质细胞灼伤机制
IF 4
Biologics : Targets & Therapy Pub Date : 2023-12-19 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S444714
Xin Ma, Junxia Jiao, Mayila Aierken, Hong Sun, Li Chen
{"title":"Hypoxia Inducible Factor-1α Through ROS/NLRP3 Pathway Regulates the Mechanism of Acute Ischemic Stroke Microglia Scorching Mechanism.","authors":"Xin Ma, Junxia Jiao, Mayila Aierken, Hong Sun, Li Chen","doi":"10.2147/BTT.S444714","DOIUrl":"https://doi.org/10.2147/BTT.S444714","url":null,"abstract":"<p><strong>Purpose: </strong>In vitro experiments explored how the hypoxia-induced factor-1α (HIF-1α) regulates the regulation of pyroptosis in microglial cells (BV 2) in acute ischemic stroke through ROS/NLRP3 pathway.</p><p><strong>Methods: </strong>The microglia acute phase oxygen-glucose deprivation/reoxygenation (OGD/R) was established, CCK-8 was applied to determine the optimal timing of intervention modeling. HIF-1α was overexpressed with stabilizer GF-4592 and HIF-1α small molecule interfering RNA (HIF-1α-siRNA), which was divided into group A (blank group), group B (OGD/R model group), group C (model+FG-4592 intervention group), group D (model+siRNA negative control group) and group E (model+HIF-1α-siRNA group). Cell proliferation of different groups was measured by CCK-8 assay. Pyroptosis and intracellular ROS levels were measured by flow cell technology. IL-18, IL-1β levels were measured by ELISA. HIF-1α, GSDMD-D, GSDMD-N, clean-Caspase-1 and NLRP3 protein expression levels were measured by Western blot. On the above experiments, ROS and NLRP3 response experiments were performed to explore how HIF-1α regulates pyroptosis through ROS/NLRP3 pathway.</p><p><strong>Results: </strong>Hypoxia for 6 h then reoxygenation for 12 h was the optimal intervention time. Compared with groups B and D, cell proliferation in group C was significantly enhanced, pyroptosis, intracellular levels of ROS, IL-18, IL-1β and the expression of GSDMD-D, GSDMD-N, clean-Caspase-1, and NLRP3 proteins were significantly decreased in group C (<i>P</i> < 0.05). However, in group E, the performance of these test indicators were exactly the opposite, and the difference was statistically significant (<i>P</i> < 0.05). Through ROS and NLRP3 response experiments, it was found that HIF-1α Inhibition of Pyroptosis by inhibiting ROS/NLRP3 pathway.</p><p><strong>Conclusion: </strong>Overexpression of HIF-1α factor can inhibit microglia pyroptosis. HIF-1α factor has an inhibitory effect on the ROS/NLRP 3 pathway, which can inhibit the pyroptotic process in microglia.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10748736/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139032159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Effect of Two Years of Secukinumab Treatment on Bone Metabolism in Patients with Radiographic Axial Spondyloarthritis: Results from Daily Clinical Practice. 塞库单抗治疗两年对轴向脊柱关节炎患者骨代谢的影响:日常临床实践的结果
IF 4
Biologics : Targets & Therapy Pub Date : 2023-12-14 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S434318
Mark Siderius, Stan C Kieskamp, Freke Wink, Frans G M Kroese, Suzanne Arends, Anneke Spoorenberg
{"title":"The Effect of Two Years of Secukinumab Treatment on Bone Metabolism in Patients with Radiographic Axial Spondyloarthritis: Results from Daily Clinical Practice.","authors":"Mark Siderius, Stan C Kieskamp, Freke Wink, Frans G M Kroese, Suzanne Arends, Anneke Spoorenberg","doi":"10.2147/BTT.S434318","DOIUrl":"10.2147/BTT.S434318","url":null,"abstract":"<p><strong>Background: </strong>Our objective was to explore bone-related outcome and bone turnover markers (BTM) during 2 years of secukinumab treatment in patients with radiographic axial spondyloarthritis (r-axSpA) in daily clinical practice.</p><p><strong>Methods: </strong>Included were consecutive r-axSpA outpatients from the Groningen Leeuwarden axSpA (GLAS) cohort treated with secukinumab for 2 years. At baseline and 2 years, spinal radiographic damage was assessed using the modified Stoke Ankylosing Spondylitis Spinal Score (mSASSS; 0-72), cervical facet joint involvement according the \"de Vlam\" scoring method (0-15) and radiographic vertebral fractures (VF) using the \"Genant\" method (grade 0-3). At all visits, BTM reflecting collagen resorption (serum type I collagen C-telopeptide; sCTX), collagen formation (procollagen type 1 N-terminal peptide; PINP) and bone mineralization (bone-specific alkaline phosphatase; BALP) were measured and expressed in Z-scores to correct for the normal influence of age and gender.</p><p><strong>Results: </strong>17 r-axSpA patients were included; 53% male, mean age was 47±15 years, mean Ankylosing Spondylitis Disease Activity Score (ASDAS) 3.9±1.2, and 53% was biological naïve. The median 2-year progression rates were 1.1 for mSASSS and 0.5 for facet joints, which was less than the smallest detectable change. One traumatic VF (grade 3) occurred. Serum levels of sCTX and PINP remained stable during secukinumab treatment and BALP decreased significantly after 2 years, with median 0-2 year change in Z-scores of +0.1, -0.4, and -1.2, respectively.</p><p><strong>Conclusion: </strong>This explorative study of r-axSpA patients treated with secukinumab in daily clinical practice showed low radiographic spinal progression during 2 years of follow-up. Collagen resorption and formation markers remained stable, whereas mineralization marker BALP decreased significantly after 2 years. Our results are in line with the results of in vitro studies demonstrating that inhibition of IL17-A resulted in suppression of osteogenic differentiation with significant decrease in mineralization.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10728592/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138798406","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Tocilizumab Vs Methotrexate in a Cohort of Patients Affected by Active GCA: A Comparative Clinical and Ultrasonographic Study. 托珠单抗与甲氨蝶呤在一组受活动性GCA影响的患者中:一项比较临床和超声研究。
IF 4
Biologics : Targets & Therapy Pub Date : 2023-12-01 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S431818
Silvia Grazzini, Edoardo Conticini, Paolo Falsetti, Miriana D'Alessandro, Jurgen Sota, Riccardo Terribili, Caterina Baldi, Claudia Fabiani, Elena Bargagli, Luca Cantarini, Bruno Frediani
{"title":"Tocilizumab Vs Methotrexate in a Cohort of Patients Affected by Active GCA: A Comparative Clinical and Ultrasonographic Study.","authors":"Silvia Grazzini, Edoardo Conticini, Paolo Falsetti, Miriana D'Alessandro, Jurgen Sota, Riccardo Terribili, Caterina Baldi, Claudia Fabiani, Elena Bargagli, Luca Cantarini, Bruno Frediani","doi":"10.2147/BTT.S431818","DOIUrl":"10.2147/BTT.S431818","url":null,"abstract":"<p><strong>Introduction: </strong>No head-to-head study has assessed the superiority of tocilizumab versus methotrexate in giant cell arteritis (GCA), and few studies have demonstrated its effectiveness in terms of ultrasonographic findings, but without a control group. The primary endpoint was to assess whether tocilizumab was superior to methotrexate in inducing normalization of US findings, whereas the secondary endpoint was to assess the effectiveness of precocious withdrawal of glucocorticoids.</p><p><strong>Methods: </strong>We prospectively enrolled all the patients with active GCA at our clinic. The inclusion criteria were clinical diagnosis of GCA; active disease; and clinical, laboratory, and US data, evaluated using the halo count (HC) and OMERACT GCA Ultrasonography Score (OGUS). Evaluations were repeated at 3, 6, and 12 months.</p><p><strong>Results: </strong>Twenty patients were treated with Tocilizumab and 9 with Methotrexate. All but three tocilizumab-treated patients achieved remission at six months, whereas at 12 months, all patients were in glucocorticoid-free remission. Up to three of the nine methotrexate patients experienced a lack of efficacy or minor relapses. Tocilizumab-treated patients showed a statistically significant difference between baseline and all follow-ups in terms of OGUS and HC, whereas the difference in the Methotrexate group was significant after 1 year. The mean glucocorticoid dosage significantly decreased in both groups. No severe adverse events or major relapses were reported.</p><p><strong>Conclusion: </strong>Our study demonstrates the superiority in terms of rapidity of a tocilizumab-based scheme over a methotrexate-based scheme in inducing clinical and US remission. Precocious withdrawal of glucocorticoids did not increase the risk of relapse.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10697083/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138497679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Serum LINC00152 and UCA1 in HCV-Induced Hepatocellular Carcinoma: Clinical Significance and Prognostic Value. 血清LINC00152和UCA1在HCV诱导的肝细胞癌中的临床意义和预后价值。
IF 4
Biologics : Targets & Therapy Pub Date : 2023-10-13 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S433872
Somaia Shehab-Eldeen, Abdallah Essa, Eman Salah Arafat, Asmaa Shaaban Sleem, Amal Abdelrasoul Alhosary, Ehab Darwish, Ali Essa, Omar Ahmed Al-Omair, Emad Ali Al-Khoufi, Abdulrhman Khaled Al Abdulqader, Ali Nada
{"title":"Serum LINC00152 and UCA1 in HCV-Induced Hepatocellular Carcinoma: Clinical Significance and Prognostic Value.","authors":"Somaia Shehab-Eldeen,&nbsp;Abdallah Essa,&nbsp;Eman Salah Arafat,&nbsp;Asmaa Shaaban Sleem,&nbsp;Amal Abdelrasoul Alhosary,&nbsp;Ehab Darwish,&nbsp;Ali Essa,&nbsp;Omar Ahmed Al-Omair,&nbsp;Emad Ali Al-Khoufi,&nbsp;Abdulrhman Khaled Al Abdulqader,&nbsp;Ali Nada","doi":"10.2147/BTT.S433872","DOIUrl":"https://doi.org/10.2147/BTT.S433872","url":null,"abstract":"<p><strong>Background: </strong>Despite significant advancements in the molecular characterization of hepatocellular carcinoma (HCC), no oncogene addiction has been discovered. Long noncoding RNAs (lncRNAs) have a lot of promise as cancer biomarkers. LINC00152 and UCA1 have shown potential as diagnostic, prognostic, and therapeutic targets for human cancers.</p><p><strong>Aim: </strong>To investigate the diagnostic and prognostic potential of serum LINC00152 and UCA1 in hepatocellular carcinoma (HCC).</p><p><strong>Methods: </strong>The expression levels of LINC00152 and UCA1 in blood samples from 120 patients (60 with HCC, 60 with liver cirrhosis) and 40 healthy subjects were assessed using real-time qRT-PCR.</p><p><strong>Results: </strong>Serum LINC00152 and UCA1 expression were considerably higher in HCC patients compared to patients with liver cirrhosis and the healthy controls (p<0.001 and p<0.001 respectively). And their expressions in the liver cirrhosis group were significantly higher than in healthy controls. Both lncRNAs performed well in the ROC analysis, distinguishing HCC patients from patients with liver cirrhosis. Higher levels of LINC00152 expression were linked to lesions in both lobes of the liver (p=0.02), while higher levels of UCA1 expression were linked to vascular invasion and the late stage (p=0.01, p=0.03 respectively). The multivariate analysis showed that a high level of LINC00152 in the blood was an independent indicator of a bad outcome for HCC patients (HR=2.23, 95% CI= 1.30-5.29, p=0.03).</p><p><strong>Conclusion: </strong>Serum LINC00152 and UCA1 expression were upregulated in patients with HCC, suggesting their use as non-invasive biomarkers for HCC. Furthermore, LINC00152 has the potential to serve as a prognostic indicator.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/68/ac/btt-17-137.PMC10581015.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49674073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment Persistence of Apremilast Among Patients with Psoriatic Arthritis. 阿普利司特在银屑病关节炎患者中的治疗持续性。
IF 4
Biologics : Targets & Therapy Pub Date : 2023-10-04 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S425693
Amir Haddad, Nili Stein, Idit Lavi, Lisa Shynkar, Irina Bergman, Ilan Feldhamer, Arnon Dov Cohen, Walid Saliba, Devy Zisman
{"title":"Treatment Persistence of Apremilast Among Patients with Psoriatic Arthritis.","authors":"Amir Haddad,&nbsp;Nili Stein,&nbsp;Idit Lavi,&nbsp;Lisa Shynkar,&nbsp;Irina Bergman,&nbsp;Ilan Feldhamer,&nbsp;Arnon Dov Cohen,&nbsp;Walid Saliba,&nbsp;Devy Zisman","doi":"10.2147/BTT.S425693","DOIUrl":"10.2147/BTT.S425693","url":null,"abstract":"<p><strong>Introduction: </strong>Persistence in drug therapy reflects treatment effectiveness and tolerability. We aim to estimate the persistence of apremilast prescribed to patients with psoriatic arthritis (PsA) and to identify characteristics associated with treatment discontinuation in a real-world setting.</p><p><strong>Methods: </strong>Patients with PsA treated with apremilast from January 2016 were identified from a large health database and followed until medication stop date (using 3-months grace period), death or the end of observation period (June 2021). Demographic data, Charlson comorbidity index and concomitant and previous use of conventional and biologic DMARDs were extracted. The reasons for drug discontinuation were manually retrieved from patient charts. Time to discontinuation was estimated using survival analysis using Kaplan-Meier functions.</p><p><strong>Results: </strong>Overall, 568 PsA patients treated with apremilast were identified. The mean age was 55.3±14.0 years, of whom 332 (58.5%) were females, 38.4% were obese (BMI>30), 75.2% had a Charlson comorbidity index>1, 24.1% were on concomitant treatment with methotrexate and 72.4% were biologic naïve. The median persistent period was 6.1,95% CI (5.2-6.9) months in which only 16.9% remained persistent on apremilast. No difference was found with regard to age, sex, socioeconomic status, ethnicity and obesity between patients who were persistent compared to patients who discontinued apremilast. Concomitant treatment with methotrexate and prior history of biologic therapy did not affect drug persistency (log rank P=0.957 and 0.082, respectively). Causes for treatment discontinuation were due to lack of skin efficacy in 19.4%, lack of joint efficacy in 33.3%, combined skin and joint inefficacy at 2.3% and due to side effects in 24.1%.</p><p><strong>Conclusion: </strong>In this large observational retrospective cohort of patients treated with apremilast, a relatively low drug persistence was observed with 6-month and 1-year survival rates of 50.3% and 31.3%, respectively. Treatment discontinuation was mainly due to joint inefficacy, advocating for more studies for proper patient selection to assure treatment effectiveness and persistency.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d9/bd/btt-17-129.PMC10560465.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41182029","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Molecular Biology Mechanisms and Emerging Therapeutics of Triple-Negative Breast Cancer. 癌症三阴性的分子生物学机制及新疗法。
IF 4
Biologics : Targets & Therapy Pub Date : 2023-09-21 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S426392
Zhiying Zhang, Rui Zhang, Donghai Li
{"title":"Molecular Biology Mechanisms and Emerging Therapeutics of Triple-Negative Breast Cancer.","authors":"Zhiying Zhang,&nbsp;Rui Zhang,&nbsp;Donghai Li","doi":"10.2147/BTT.S426392","DOIUrl":"https://doi.org/10.2147/BTT.S426392","url":null,"abstract":"<p><p>Triple-negative breast cancer (TNBC) is an aggressive subtype of breast cancer that is conventionally characterized by the absence of estrogen receptor (ER), progesterone receptor (PR), and human epidermal growth factor receptor-2 (HER2), accounting for approximately 15-20% of all breast cancers. Compared to other molecular phenotypes, TNBC is typically associated with high malignancy and poor prognosis. Cytotoxic agents have been the mainstay of treatment for the past few decades due to the lack of definitive targets and limited therapeutic interventions. However, recent developments have demonstrated that TNBC has peculiar molecular classifications and biomarkers, which provide the possibility of evolving treatment from basic cytotoxic chemotherapy to an expanding domain of targeted therapies. This review presents a framework for understanding the current clinical experience surrounding molecular biology mechanisms in TNBC (Figure 1). Including immunotherapy, polymerase (PARP) and PI3K/AKT pathway inhibitors, antibody-drug conjugates, and androgen receptor (AR) blockade. Additionally, the role of miRNA therapeutics targeting TNBC and potential strategies targeting cancer stem cells (CSCs) are discussed and highlighted. As more and more treatments arise on the horizon, we believe that patients with TNBC will have a new sense of hope.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/85/41/btt-17-113.PMC10520847.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41096964","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Demonstration of Safety in Wild Type Mice of npFOXF1, a Novel Nanoparticle-Based Gene Therapy for Alveolar Capillary Dysplasia with Misaligned Pulmonary Veins. 基于纳米粒子的新型基因疗法 npFOXF1 对肺泡毛细血管发育不良伴肺静脉错位症野生型小鼠的安全性证明
IF 4
Biologics : Targets & Therapy Pub Date : 2023-03-20 eCollection Date: 2023-01-01 DOI: 10.2147/BTT.S400006
Fatemeh Kohram, Zicheng Deng, Yufang Zhang, Abid A Al Reza, Enhong Li, Olena A Kolesnichenko, Samriddhi Shukla, Vladimir Ustiyan, Jose Gomez-Arroyo, Anusha Acharya, Donglu Shi, Vladimir V Kalinichenko, Alan P Kenny
{"title":"Demonstration of Safety in Wild Type Mice of npFOXF1, a Novel Nanoparticle-Based Gene Therapy for Alveolar Capillary Dysplasia with Misaligned Pulmonary Veins.","authors":"Fatemeh Kohram, Zicheng Deng, Yufang Zhang, Abid A Al Reza, Enhong Li, Olena A Kolesnichenko, Samriddhi Shukla, Vladimir Ustiyan, Jose Gomez-Arroyo, Anusha Acharya, Donglu Shi, Vladimir V Kalinichenko, Alan P Kenny","doi":"10.2147/BTT.S400006","DOIUrl":"10.2147/BTT.S400006","url":null,"abstract":"<p><strong>Introduction: </strong>Alveolar Capillary Dysplasia with Misaligned Pulmonary Veins (ACDMPV) is a fatal congenital disease resulting from a pulmonary vascular endothelial deficiency of FOXF1, producing abnormal morphogenesis of alveolar capillaries, malpositioned pulmonary veins and disordered development of lung lobes. Affected neonates suffer from cyanosis, severe breathing insufficiency, pulmonary hypertension, and death typically within days to weeks after birth. Currently, no treatment exists for ACDMPV, although recent murine research in the Kalinichenko lab demonstrates nanoparticle delivery improves survival and reconstitutes normal alveolar-capillary architecture. The aim of the present study is to investigate the safety of intravenous administration of FOXF1-expressing PEI-PEG nanoparticles (npFOXF1), our pioneering treatment for ACDMPV.</p><p><strong>Methods: </strong>npFOXF1 was constructed, validated, and subsequently administered in a single dose to postnatal day 14 (P14) mice via retro-orbital injection. Biochemical, serologic, and histologic safety were monitored at postnatal day 16 (P16) and postnatal day 21 (P21).</p><p><strong>Results: </strong>With treatment we observed no lethality, and the general condition of mice revealed no obvious abnormalities. Serum chemistry, whole blood, and histologic toxicity was assayed on P16 and P21 and revealed no abnormality.</p><p><strong>Discussion: </strong>In conclusion, npFOXF1 has a very good safety profile and combined with preceding studies showing therapeutic efficacy, npFOXF1 can be considered as a good candidate therapy for ACDMPV in human neonates.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/bb/46/btt-17-43.PMC10031269.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9587136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Significance of Interleukin 23 in Systemic Lupus Patients: Relation to Disease Activity and Damage Indices. 白细胞介素23在系统性狼疮患者中的意义:与疾病活动性和损伤指标的关系。
IF 4
Biologics : Targets & Therapy Pub Date : 2023-01-01 DOI: 10.2147/BTT.S389021
Maysa M Haroon, Gehan A Hegazy, Mohammed A Hassanien, Olfat Shaker, Wafaa H Hussein
{"title":"Significance of Interleukin 23 in Systemic Lupus Patients: Relation to Disease Activity and Damage Indices.","authors":"Maysa M Haroon,&nbsp;Gehan A Hegazy,&nbsp;Mohammed A Hassanien,&nbsp;Olfat Shaker,&nbsp;Wafaa H Hussein","doi":"10.2147/BTT.S389021","DOIUrl":"https://doi.org/10.2147/BTT.S389021","url":null,"abstract":"<p><strong>Background: </strong>Dysregulation of both cellular and humoral immune responses is central in systemic lupus erythematosus (SLE) pathogenetic mechanisms. Proinflammatory cytokines, such as interleukin 23 (IL23), and their roles in promoting such dysregulation have recently been highly considered. This research compared IL23 serum levels in 85 Egyptian SLE patients and 85 healthy controls. Then, IL23 level was correlated to various SLE disease parameters, disease activity, and damage indices.</p><p><strong>Results: </strong>IL23 serum levels were significantly elevated in SLE patients versus healthy individuals. Furthermore, IL23 levels were positively correlated with SLE disease activity index (SLEDAI) and were positively correlated with arthritis, seizures, consumption of complements (C3, C4), and with parameters of nephritis (hematuria, pyuria, casts, and proteinuria). A positive correlation was also found between IL23 levels and oral prednisolone dose.</p><p><strong>Conclusion: </strong>IL23 has higher levels in the serum of SLE patients, and is correlated to activity of the disease, especially lupus nephritis. Further researchis needed to explore its exact role in SLE pathogenesis and whether it can be considered a potential biomarker or therapeutic target in SLE.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/7a/46/btt-17-1.PMC9868139.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10622308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Effectiveness of RCTs Pooling Evidence on Mesenchymal Stem Cell (MSC) Therapeutic Applications During COVID-19 Epidemic: A Systematic Review. 在COVID-19流行期间间充质干细胞(MSC)治疗应用的随机对照试验汇集证据的有效性:系统综述
IF 4
Biologics : Targets & Therapy Pub Date : 2023-01-01 DOI: 10.2147/BTT.S404421
Usha Rani Kandula, Addisu Dabi Wake
{"title":"Effectiveness of RCTs Pooling Evidence on Mesenchymal Stem Cell (MSC) Therapeutic Applications During COVID-19 Epidemic: A Systematic Review.","authors":"Usha Rani Kandula,&nbsp;Addisu Dabi Wake","doi":"10.2147/BTT.S404421","DOIUrl":"https://doi.org/10.2147/BTT.S404421","url":null,"abstract":"<p><strong>Background: </strong>Global pandemic identified as coronavirus disease 2019 (COVID-19) has resulted in a variety of clinical symptoms, from asymptomatic carriers to those with severe acute respiratory distress syndrome (SARS) and moderate upper respiratory tract symptoms (URTS). This systematic review aimed to determine effectiveness of stem cell (SC) applications among COVID-19 patients.</p><p><strong>Methods: </strong>Multiple databases of PubMed, EMBASE, Science Direct, Google Scholar, Scopus, Web of Science, and Cochrane Library were used. Studies were screened, chosen, and included in this systematic review using Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 flowchart diagram and PRISMA checklist. Included studies' quality was assessed employing Critical Appraisal Skills Programme (CASP) quality evaluation criteria for 14 randomized controlled trials (RCTs).</p><p><strong>Results: </strong>Fourteen RCTs were performed between the years of 2020 to 2022, respectively, with a sample size n = 574 (treatment group (n = 318); control group (n = 256)) in multiple countries of Indonesia, Iran, Brazil, Turkey, China, Florida, UK, and France. The greatest sample size reported from China among 100 COVID-19 patients, while the lowest sample of 9 COVID-19 patients from Jakarta, Indonesia, and the patient's age ranges from 18 to 69 years. Studies applied to the type of SC were \"Umbilical cord MSCs, MSCs secretome, MSCs, Placenta-derived MSCs, Human immature dental pulp SC, DW-MSC infusion, Wharton Jelly-derived MSCs\". The injected therapeutic dose was 1 × 10<sup>6</sup> cells/kg, 1 × 10<sup>7</sup> cells/kg, 1 × 10<sup>5</sup> cells/kg, and 1 million cells/kg as per the evidence from the different studies. Studies focused on demographic variables, clinical symptoms, laboratory tests, Comorbidities, respiratory measures, concomitant therapies, Sequential Organ Failure Assessment score, mechanical ventilation, body mass index, adverse events, inflammatory markers, and PaO<sub>2</sub>/FiO<sub>2</sub> ratio were all recorded as study characteristics.</p><p><strong>Conclusion: </strong>Clinical evidence on MSC's therapeutic applications during COVID-19 pandemic has proven to be a promising therapy for COVID-19 patient recovery with no consequences and applied as a routine treatment for challenging ailments.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/cc/94/btt-17-85.PMC10202141.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9516657","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信