The Journal of dermatological treatment最新文献_第9页

Paradoxical skin lesions induced by IL-17 inhibitors in SAPHO syndrome. SAPHO综合征中IL-17抑制剂诱发的矛盾性皮肤损伤。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2023-12-28 DOI: 10.1080/09546634.2023.2295239

Yuan Li, Fanzhang Meng, Haixu Jiang, Qiuwei Peng, Jingyuan Fu, Dan Yin, Xiujuan Hou, Chen Li

引用次数: 0

A systematic review and meta-analysis of efficacy and safety of compound glycyrrhizin combined with second-generation non-sedated antihistamine for the treatment of chronic urticaria. 复方甘草酸苷联合第二代非镇静抗组胺药治疗慢性荨麻疹的疗效和安全性的系统综述和荟萃分析。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-01-03 DOI: 10.1080/09546634.2023.2299597

Sijue Chen, Wei Cao, Xianjun Xiao, Lu Wang, Renhong Wan, Zihao Zou, Qian Yang, Ying Li

{"title":"A systematic review and meta-analysis of efficacy and safety of compound glycyrrhizin combined with second-generation non-sedated antihistamine for the treatment of chronic urticaria.","authors":"Sijue Chen, Wei Cao, Xianjun Xiao, Lu Wang, Renhong Wan, Zihao Zou, Qian Yang, Ying Li","doi":"10.1080/09546634.2023.2299597","DOIUrl":"10.1080/09546634.2023.2299597","url":null,"abstract":"Background: Chronic urticaria (CU) is a prevalent dermatologic disease that negatively affects life, current therapies remain suboptimal. Hence, there is an urgent need to identify effective and safe treatment.Objective: Assess the efficacy and safety of compound glycyrrhizin (CG) combined with second-generation nonsedated antihistamine for the treatment of CU.Methods: Nine databases were queried to screen RCTs related. Two reviewers independently assessed the risk of bias using Cochrane Collaboration. Primary objective was the total efficiency rate, while secondary was rate of recurrence, adverse events, and cure. Statistical analyses using Review Manager 5.4 and Stata17.Results: Twenty-four RCTs were identified. Significant differences were noted in rate of total efficiency (n = 2649, RR = 1.36, 95%CI:1.30-1.43, p < 0.00001), cure (n = 2649, RR = 1.54, 95%CI:1.42-1.66, p < 0.00001) and recurrence (n = 446, RR = 0.34, 95%CI:0.20-0.58, p < 0.00001) between the combination of CG with second-generation non-sedated antihistamine and antihistamine monotherapy. Contrastingly, adverse events rate (n = 2317, RR = 0.76, 95% CI:0.59-0.97, p = 0.03) was comparable between the two groups. Our results indicated that CG combined with second-generation non-sedated antihistamine could significantly mitigate the symptoms in CU compared with antihistamine monotherapy. No serious adverse events were reported.Conclusions: CG combined with second-generation nonsedated antihistamine is effective for CU. Nevertheless, higher-quality studies are warranted to validate our results.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2299597"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139081192","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Cost per response analysis of deucravacitinib versus apremilast and first-line biologics among patients with moderate to severe plaque psoriasis in the United States. 美国中重度斑块状银屑病患者服用 deucravacitinib 与 apremilast 和一线生物制剂的每次应答成本分析。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-06-24 DOI: 10.1080/09546634.2024.2366503

Sang Hee Park, Mark Lambton, Jordana Schmier, Sara Hovland, Keith Wittstock, Vardhaman Patel

{"title":"Cost per response analysis of deucravacitinib versus apremilast and first-line biologics among patients with moderate to severe plaque psoriasis in the United States.","authors":"Sang Hee Park, Mark Lambton, Jordana Schmier, Sara Hovland, Keith Wittstock, Vardhaman Patel","doi":"10.1080/09546634.2024.2366503","DOIUrl":"https://doi.org/10.1080/09546634.2024.2366503","url":null,"abstract":"Background: Understanding the economic value of deucravacitinib and apremilast could assist treatment decision-making for patients with moderate to severe plaque psoriasis.Objective: This study compared the cost per response (CPR) for US patients initiating deucravacitinib versus apremilast for moderate to severe plaque psoriasis.Methods: A CPR model using pharmacy and administration costs was developed from a US payer perspective. Response was defined as a 75% reduction from baseline in Psoriasis Area and Severity Index (PASI 75) at weeks 16 and 24. Long-term response was defined as the cumulative benefit over 52 weeks, measured as area under the curve; subsequent treatment was included. Scenario analyses explored varying the efficacy measure or choices of subsequent treatments and limiting discontinuation.Results: The CPR for deucravacitinib versus apremilast was lower at 16 weeks (difference: -$3796 [95% confidence interval (CI): -$6140 to -$1659]) and 24 weeks (difference: -$12,784 [95% CI: -$16,674 to -$9369]). At 52 weeks, the cost per cumulative benefit was lower for patients who initiated deucravacitinib, regardless of initial treatment period duration (16 or 24 weeks).Conclusions: Scenario analyses found mainly consistent results. This study showed that the CPR is lower when initiating deucravacitinib versus apremilast in moderate to severe plaque psoriasis.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2366503"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141447907","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Frequency and outcomes of treatment dose escalation with biologics in moderate-to-severe psoriasis: a Swedish register study. 中度至重度银屑病患者使用生物制剂进行剂量升级治疗的频率和结果：一项瑞典登记研究。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-09-04 DOI: 10.1080/09546634.2024.2398170

Axel Svedbom, Christina Wennerström, Fredrik Hjelm, Anna Tjärnlund, Mona Ståhle

{"title":"Frequency and outcomes of treatment dose escalation with biologics in moderate-to-severe psoriasis: a Swedish register study.","authors":"Axel Svedbom, Christina Wennerström, Fredrik Hjelm, Anna Tjärnlund, Mona Ståhle","doi":"10.1080/09546634.2024.2398170","DOIUrl":"https://doi.org/10.1080/09546634.2024.2398170","url":null,"abstract":"Background: The advent of biosimilars may increase the frequency of dose escalation with biologics in psoriasis.Objective: To explore the frequency and outcomes of dose escalation with adalimumab etanercept, and ustekinumab.Methods: Data were extracted from DermaReg-Pso, a psoriasis register in Stockholm, Sweden. The main exposure was treatment, and the main outcome was dose escalation. We describe outcomes with dose escalation by estimating drug survival and changes in the Psoriasis Area and Severity Index (PASI).Results: 554 patients had 946 treatment episodes with adalimumab, etanercept, or ustekinumab. The cumulative incidence of dose escalation was 4.1 per 100 treatment years. The Hazard Ratios (HRs) for dose escalation with ustekinumab vs adalimumab and ustekinumab vs etanercept were 1.93 (95% CI: 1.25-2.98), and 2.20 (95% CI: 1.42-3.41), respectively. After dose escalation, the HRs for treatment discontinuation with adalimumab and etanercept compared with ustekinumab were 3.10 (95% CI: 1.56-6.18) and 7.15 (95% CI: 3.96-12.94), respectively. PASI was higher after compared to before dose escalation for etanercept (p = 0.036), but not for adalimumab (p = 0.832) or ustekinumab (p = 0.300).Conclusions: Dose escalation was comparatively more frequent with ustekinumab than with adalimumab or etanercept; however, treatment discontinuation after dose escalation was more common with adalimumab and etanercept than ustekinumab.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2398170"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142127793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Disease burden and patient characteristics associated with systemic therapy utilization among adults with atopic dermatitis: data from CorEvitas Atopic Dermatitis Registry. 与特应性皮炎成人患者使用系统疗法相关的疾病负担和患者特征：来自 CorEvitas 特应性皮炎登记处的数据。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-09-25 DOI: 10.1080/09546634.2024.2396382

Jonathan I Silverberg, Linda Stein Gold, Seemal Desai, Alexandra Golant, Douglas DiRuggiero, D Christian Fenske, Alvin Li, Zach Dawson, Yolanda Muñoz Maldonado, Kaylee Ho, Kayla Callahan, Eric L Simpson

{"title":"Disease burden and patient characteristics associated with systemic therapy utilization among adults with atopic dermatitis: data from CorEvitas Atopic Dermatitis Registry.","authors":"Jonathan I Silverberg, Linda Stein Gold, Seemal Desai, Alexandra Golant, Douglas DiRuggiero, D Christian Fenske, Alvin Li, Zach Dawson, Yolanda Muñoz Maldonado, Kaylee Ho, Kayla Callahan, Eric L Simpson","doi":"10.1080/09546634.2024.2396382","DOIUrl":"https://doi.org/10.1080/09546634.2024.2396382","url":null,"abstract":"Background: The decision to initiate advanced systemics in patients with atopic dermatitis (AD) is complex.Objectives: To explore disease burden and clinical characteristics of patients with moderate-to-severe AD and identify characteristics associated with initiating new systemics.Methods: Data from prospective, longitudinal, non-interventional CorEvitas AD Registry were evaluated. Differences in demographic and clinical characteristics, comorbidities, disease severity (vIGA-AD™; body surface area (BSA); Eczema Area and Severity Index (EASI); SCORing AD [SCORAD]), and patient-reported outcomes (PROs) were assessed between systemic and non-systemic therapy groups.Results: Of 883 patients, 673 were newly prescribed systemics and 210 were not. Non-systemic therapy group had higher than expected rates of severe disease at enrollment based on vIGA-AD = 4 (39%), mean BSA involvement (31%), and mean EASI (19). PROs for non-systemic therapy group indicated elevated burden from AD on quality of life and poor disease control. SCORAD, peak pruritus in the past 24 h, history of biologics, and facial pallor, were significantly associated with initiation of systemics at enrollment.Conclusion: While disease burden likely influences the initiation of systemic therapy, many patients with significant burden are not treated with systemics for unclear reasons. Further research is needed to identify other factors, beyond disease severity, that influence this decision.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2396382"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142335571","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Biologics and oral systemic treatment preferences in patients and physicians for moderate-to-severe atopic dermatitis: a discrete choice experiment in the United Kingdom and Germany. 中重度特应性皮炎患者和医生对生物制剂和口服系统治疗的偏好：英国和德国的离散选择实验。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-10-27 DOI: 10.1080/09546634.2024.2417966

Nicholas Durno, Pablo Arija, Krystallia Pantiri, Marieke Heisen, Marco Boeri, Josef Paris, Katrin Jack, Olivier Chambenoit, Ramkumar Subramanian, Jorge Puelles, Elly Stolk, Ben van Hout, Jonathan I Silverberg

{"title":"Biologics and oral systemic treatment preferences in patients and physicians for moderate-to-severe atopic dermatitis: a discrete choice experiment in the United Kingdom and Germany.","authors":"Nicholas Durno, Pablo Arija, Krystallia Pantiri, Marieke Heisen, Marco Boeri, Josef Paris, Katrin Jack, Olivier Chambenoit, Ramkumar Subramanian, Jorge Puelles, Elly Stolk, Ben van Hout, Jonathan I Silverberg","doi":"10.1080/09546634.2024.2417966","DOIUrl":"https://doi.org/10.1080/09546634.2024.2417966","url":null,"abstract":"Background: As the available treatments for moderate-to-severe atopic dermatitis (AD) expand, understanding patient and physician preferences becomes crucial for informed decision-making.Objective: To quantify patient and physician preferences for biologics and oral systemic AD treatment attributes.Materials and methods: We conducted a cross-sectional, online discrete choice experiment (DCE) involving 306 AD patients and 206 physicians throughout the United Kingdom and Germany. Qualitative interviews identified the key attributes for inclusion in the DCE. Each choice task comprised two hypothetical patient profiles. Data were analyzed using a random-parameters logit model.Results: Results indicated a significant emphasis on efficacy, with reducing sleep disturbance and itch ranking first and second among patients, and the reverse for physicians. Time to itch relief was the third most important efficacy attribute for both groups, but relatively more important for patients than for physicians. For both groups, the risk of eye problems was the most important safety concern of those included. Mode of administration was not of great importance compared to efficacy and safety attributes.Conclusions: Our findings suggest patients prioritize sleep disturbance, an attribute not captured in prior preference studies in AD, time to itch relief and itch. These findings emphasize the importance of addressing sleep-related issues, whilst also targeting fast itch control, to enhance patients' well-being.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2417966"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142515607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparison of safety profile in patients with atopic dermatitis treated with dupilumab or conventional systemic treatment: real world data from the US network. 特应性皮炎患者接受杜匹单抗治疗或传统系统治疗的安全性比较：来自美国网络的真实数据。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-11-03 DOI: 10.1080/09546634.2024.2421429

Henner Zirpel, Ralf J Ludwig, Henning Olbrich, Khalaf Kridin, Sascha Ständer, Diamant Thaçi

{"title":"Comparison of safety profile in patients with atopic dermatitis treated with dupilumab or conventional systemic treatment: real world data from the US network.","authors":"Henner Zirpel, Ralf J Ludwig, Henning Olbrich, Khalaf Kridin, Sascha Ständer, Diamant Thaçi","doi":"10.1080/09546634.2024.2421429","DOIUrl":"https://doi.org/10.1080/09546634.2024.2421429","url":null,"abstract":"Background: Safety of dupilumab in atopic dermatitis (AD) was investigated in randomized controlled trials (RCT). However, head-to-head trials comparing with conventional systemic drugs are lacking and large real-world data on the long-term safety profile as compared are scarce.Objective: To compare long-term safety profile of dupilumab with conventional systemic drugs used in the management of moderate to severe AD.Methods: Data from electronic health records of AD patients treated with either dupilumab, azathioprine, Cyclosporine A, mycophenolate mofetil, methotrexate, or oral glucocorticoids were retrieved from the TriNetX US Collaborative Network. Risks of adverse events and new onset of type-2-inflammatory diseases within 5 years after treatment initiation was investigated.Results: 5 propensity-matched cohorts, up to 18,708 individuals per cohort, were created. Dupilumab treatment displayed reduced risk for diseases of the circulatory, the upper respiratory, and the musculoskeletal system, infections, and type 2 diseases as compared to all other treatment options. In contrast risk for conjunctivitis was increased in dupilumab treated patients as compared to mycophenolate mofetil and methotrexate.Conclusion: Here presented data indicates that treatment with dupilumab for AD has reduced risk for adverse effects of conventional systemic drugs and thus might be safer. Obtained data should be verified in prospective studies.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2421429"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142570856","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Tranexamic acid as a therapeutic option for melasma management: meta-analysis and systematic review of randomized controlled trials. 氨甲环酸作为治疗黄褐斑的一种选择：随机对照试验的荟萃分析和系统回顾。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-06-06 DOI: 10.1080/09546634.2024.2361106

Retaj Calacattawi, Mohammed Alshahrani, Maryam Aleid, Fatimah Aleid, Khalid Basamih, Ghada Alsugair, Raghad Alqahtani, Noor AlKhabbaz, Yaser Algaidi, Latifa Alrakayan, Abdulaziz Almohanna, Afnan Madkhali, Shaima Aljohani, Naif Alotibi

{"title":"Tranexamic acid as a therapeutic option for melasma management: meta-analysis and systematic review of randomized controlled trials.","authors":"Retaj Calacattawi, Mohammed Alshahrani, Maryam Aleid, Fatimah Aleid, Khalid Basamih, Ghada Alsugair, Raghad Alqahtani, Noor AlKhabbaz, Yaser Algaidi, Latifa Alrakayan, Abdulaziz Almohanna, Afnan Madkhali, Shaima Aljohani, Naif Alotibi","doi":"10.1080/09546634.2024.2361106","DOIUrl":"10.1080/09546634.2024.2361106","url":null,"abstract":"Purpose: This study aimed to evaluate the efficacy of tranexamic acid (TXA) in treating melasma through a meta-analysis and systematic review of randomized controlled trials (RCTs). The study focused on identifying associated adverse effects and comparing TXA's effectiveness with other melasma treatments.Materials and methods: Following PROSPERO and PRISMA guidelines, an extensive electronic search was conducted across four databases for RCTs on TXA use in melasma. Inclusion criteria encompassed full-text English articles with specific outcome measures, while studies with high bias risk or non-English publications were excluded. Data were extracted from 22 relevant studies and analyzed using the RevMan software, with heterogeneity identified using I² statistics and forest plots.Results: A total of 22 studies with 1280 patients were included. TXA was administered orally, topically, or via injection, with treatment durations ranging from 8 weeks to nearly 2 years. TXA significantly reduced melasma severity, evidenced by reductions in MASI, mMASI, MI, and hemi-MASI scores. Oral TXA showed the most substantial decrease in MASI scores, followed by injections and topical applications. However, studies exhibited high heterogeneity, particularly in combined treatments. Adverse effects included gastrointestinal discomfort, skin irritation, and menstrual irregularities.Conclusions: TXA is effective in treating melasma, either alone or combined with other treatments. Despite significant reductions in melasma severity, further research is necessary to standardize TXA administration methods and address long-term effects. The high heterogeneity observed suggests a need for more consistent treatment protocols.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2361106"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141285696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The development and initial evaluation of conversation cards for optimizing consultations for patients with atopic dermatitis. 开发并初步评估用于优化特应性皮炎患者咨询的对话卡。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-07-09 DOI: 10.1080/09546634.2024.2376268

Anna Sophie Belling Krontoft, Kirsten Lomborg, Lone Skov

{"title":"The development and initial evaluation of conversation cards for optimizing consultations for patients with atopic dermatitis.","authors":"Anna Sophie Belling Krontoft, Kirsten Lomborg, Lone Skov","doi":"10.1080/09546634.2024.2376268","DOIUrl":"https://doi.org/10.1080/09546634.2024.2376268","url":null,"abstract":"Purpose: Patients with atopic dermatitis (AD) require both skills and support to effectively manage life with the disease. Here, we developed an agenda-setting tool for consultations with patients with AD to establish a collaborative agenda that enhances patient involvement and prioritizes on self-management support.Materials and methods: Using the design thinking process, we included 64 end-users (patients and healthcare professionals (HCPs)) across the different phases of design thinking. We identified seven overall categories that patients find important to discuss during consultations, which informed the development of a tool for co-creating a consultation agenda (conversation cards, CCs).Results: Through iterative user testing of the CCs, patients perceived the cards as both inspiring and an invitation from HCPs to openly discuss their needs during consultations. Healthcare professionals have found the CCs easy to use, despite the disruption to the typical consultation process.Conclusion: In summary, the CCs provide a first-of-its-kind agenda-setting tool for patients with AD. They offer a simple and practical method to establishing a shared agenda that focuses on the patients' needs and are applicable within real-world clinical settings.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2376268"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141565498","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa. 治疗大疱性表皮松解症的新兴 DNA 和 RNA 编辑策略。

The Journal of dermatological treatment Pub Date : 2024-12-01 Epub Date: 2024-08-18 DOI: 10.1080/09546634.2024.2391452

Ulrich Koller, Johann W Bauer

{"title":"Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa.","authors":"Ulrich Koller, Johann W Bauer","doi":"10.1080/09546634.2024.2391452","DOIUrl":"https://doi.org/10.1080/09546634.2024.2391452","url":null,"abstract":"Background: Epidermolysis bullosa (EB) is a clinically-heterogeneous genodermatosis with severe manifestations in the skin and other organs. The significant burden this condition places on patients justifies the development of gene therapeutic strategies targeting the genetic cause of the disease.Methods: Emerging RNA and DNA editing tools have shown remarkable advances in efficiency and safety. Applicable both in ex vivo- and in vivo settings, these gene therapeutics based on gene replacement or editing are either at the pre-clinical or clinical stage.Results: The recent landmark FDA approvals for gene editing based on CRISPR/Cas9, along with the first FDA-approved redosable in vivo gene replacement therapy for EB, will invigorate ongoing research efforts, increasing the likelihood of achieving local cure via CRISPR-based technologies in the near future.Conclusions: This review discusses the status quo of current gene therapeutics that act at the level of RNA or DNA, all with the common aim of improving the quality of life for EB patients.","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2391452"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142001633","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0