Advanced Therapeutics最新文献_第3页

Aptamer-Guided CRISPR Systems Delivery: Precision Targeting Strategies, Translational Barriers, and Therapeutic Potential 适体引导的CRISPR系统递送：精确靶向策略，翻译障碍和治疗潜力

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-25 DOI: 10.1002/adtp.202500395

Yousef Ijjeh, Nowar Alsarayreh, Dana Alqudah, Nirmeen Elzogheir, Ali Abuhaliema, Mohammad R. Shomali, Alqassem Abuarqoub, Mohammad IA Ahmad, Sharif Abdelghany, Alaaldin M. Alkilany, Walhan Alshaer

{"title":"Aptamer-Guided CRISPR Systems Delivery: Precision Targeting Strategies, Translational Barriers, and Therapeutic Potential","authors":"Yousef Ijjeh, Nowar Alsarayreh, Dana Alqudah, Nirmeen Elzogheir, Ali Abuhaliema, Mohammad R. Shomali, Alqassem Abuarqoub, Mohammad IA Ahmad, Sharif Abdelghany, Alaaldin M. Alkilany, Walhan Alshaer","doi":"10.1002/adtp.202500395","DOIUrl":"https://doi.org/10.1002/adtp.202500395","url":null,"abstract":"<div>\u0000 \u0000 <p>The CRISPR system has revolutionized genome editing due to its capability to modify genetic material with precision, programmability, and potential for treating disorders, cancers, and autoimmune diseases. Its use in the clinic is constrained by delivery inefficiencies, off-target effects, and immunogenicity. Aptamers have been touted as a solution to overcoming these constraints, as they are short, single-stranded nucleic acids that possess high specificity and affinity for cellular targets. This review highlights the convergence of aptamer technology and CRISPR-Cas9 delivery, with the focus on conjugation chemistries to engineer targeted delivery systems. This review discusses various aptamer-functionalized platforms, including lipid nanoparticles, polymeric carriers, gold nanoparticles, and DNA/RNA nanostructures, and their potential for cellular uptake, endosomal release, and site-specific genome editing. Therapeutic uses in cancer gene editing, immune cell engineering, and gene correction of genetic diseases are discussed, along with the translational hurdles, including aptamer and CRISPR components stability, immunogenicity, off-targeting, and manufacturing. Finally, future directions include the combination of aptamer-directed systems with next-generation genome sequencing, artificial intelligence-aided aptamer design, and addressing the regulatory challenges associated with clinical use. By leveraging the molecular selectivity of aptamers, this strategy could lead to a family of safe, efficacious, and targeted gene therapeutics.</p>\u0000 </div>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 3","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147568835","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Aptamer-Guided CRISPR Systems Delivery: Precision Targeting Strategies, Translational Barriers, and Therapeutic Potential 适体引导的CRISPR系统递送：精确靶向策略，翻译障碍和治疗潜力

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-25 DOI: 10.1002/adtp.202500395

Yousef Ijjeh, Nowar Alsarayreh, Dana Alqudah, Nirmeen Elzogheir, Ali Abuhaliema, Mohammad R. Shomali, Alqassem Abuarqoub, Mohammad IA Ahmad, Sharif Abdelghany, Alaaldin M. Alkilany, Walhan Alshaer

{"title":"Aptamer-Guided CRISPR Systems Delivery: Precision Targeting Strategies, Translational Barriers, and Therapeutic Potential","authors":"Yousef Ijjeh, Nowar Alsarayreh, Dana Alqudah, Nirmeen Elzogheir, Ali Abuhaliema, Mohammad R. Shomali, Alqassem Abuarqoub, Mohammad IA Ahmad, Sharif Abdelghany, Alaaldin M. Alkilany, Walhan Alshaer","doi":"10.1002/adtp.202500395","DOIUrl":"https://doi.org/10.1002/adtp.202500395","url":null,"abstract":"<div>\u0000 \u0000 <p>The CRISPR system has revolutionized genome editing due to its capability to modify genetic material with precision, programmability, and potential for treating disorders, cancers, and autoimmune diseases. Its use in the clinic is constrained by delivery inefficiencies, off-target effects, and immunogenicity. Aptamers have been touted as a solution to overcoming these constraints, as they are short, single-stranded nucleic acids that possess high specificity and affinity for cellular targets. This review highlights the convergence of aptamer technology and CRISPR-Cas9 delivery, with the focus on conjugation chemistries to engineer targeted delivery systems. This review discusses various aptamer-functionalized platforms, including lipid nanoparticles, polymeric carriers, gold nanoparticles, and DNA/RNA nanostructures, and their potential for cellular uptake, endosomal release, and site-specific genome editing. Therapeutic uses in cancer gene editing, immune cell engineering, and gene correction of genetic diseases are discussed, along with the translational hurdles, including aptamer and CRISPR components stability, immunogenicity, off-targeting, and manufacturing. Finally, future directions include the combination of aptamer-directed systems with next-generation genome sequencing, artificial intelligence-aided aptamer design, and addressing the regulatory challenges associated with clinical use. By leveraging the molecular selectivity of aptamers, this strategy could lead to a family of safe, efficacious, and targeted gene therapeutics.</p>\u0000 </div>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 3","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147568836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Issue Information (Adv. Therap. 3/2026) 发布信息（ad . Therap. 3/2026）

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-25 DOI: 10.1002/adtp.70136

引用次数: 0

Aptamer-Guided CRISPR Systems Delivery: Precision Targeting Strategies, Translational Barriers, and Therapeutic Potential 适体引导的CRISPR系统递送：精确靶向策略，翻译障碍和治疗潜力

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-25 DOI: 10.1002/adtp.202500395

Yousef Ijjeh, Nowar Alsarayreh, Dana Alqudah, Nirmeen Elzogheir, Ali Abuhaliema, Mohammad R. Shomali, Alqassem Abuarqoub, Mohammad IA Ahmad, Sharif Abdelghany, Alaaldin M. Alkilany, Walhan Alshaer

{"title":"Aptamer-Guided CRISPR Systems Delivery: Precision Targeting Strategies, Translational Barriers, and Therapeutic Potential","authors":"Yousef Ijjeh, Nowar Alsarayreh, Dana Alqudah, Nirmeen Elzogheir, Ali Abuhaliema, Mohammad R. Shomali, Alqassem Abuarqoub, Mohammad IA Ahmad, Sharif Abdelghany, Alaaldin M. Alkilany, Walhan Alshaer","doi":"10.1002/adtp.202500395","DOIUrl":"10.1002/adtp.202500395","url":null,"abstract":"<div>\u0000 \u0000 <p>The CRISPR system has revolutionized genome editing due to its capability to modify genetic material with precision, programmability, and potential for treating disorders, cancers, and autoimmune diseases. Its use in the clinic is constrained by delivery inefficiencies, off-target effects, and immunogenicity. Aptamers have been touted as a solution to overcoming these constraints, as they are short, single-stranded nucleic acids that possess high specificity and affinity for cellular targets. This review highlights the convergence of aptamer technology and CRISPR-Cas9 delivery, with the focus on conjugation chemistries to engineer targeted delivery systems. This review discusses various aptamer-functionalized platforms, including lipid nanoparticles, polymeric carriers, gold nanoparticles, and DNA/RNA nanostructures, and their potential for cellular uptake, endosomal release, and site-specific genome editing. Therapeutic uses in cancer gene editing, immune cell engineering, and gene correction of genetic diseases are discussed, along with the translational hurdles, including aptamer and CRISPR components stability, immunogenicity, off-targeting, and manufacturing. Finally, future directions include the combination of aptamer-directed systems with next-generation genome sequencing, artificial intelligence-aided aptamer design, and addressing the regulatory challenges associated with clinical use. By leveraging the molecular selectivity of aptamers, this strategy could lead to a family of safe, efficacious, and targeted gene therapeutics.</p>\u0000 </div>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 3","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147568567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Issue Information (Adv. Therap. 3/2026) 发布信息（ad . Therap. 3/2026）

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-25 DOI: 10.1002/adtp.70136

引用次数: 0

Long-Acting Hydrogel-Based Depot Formulations of Tirzepatide and Semaglutide for the Management of Type 2 Diabetes and Weight 替西帕肽和西马鲁肽的长效水凝胶储备制剂用于治疗2型糖尿病和体重

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-22 DOI: 10.1002/adtp.202500329

Andrea I. d'Aquino, Changxin Dong, Leslee T. Nguyen, Jerry Yan, Carolyn K. Jons, Olivia M. Saouaf, Ye Eun Song, Noah Eckman, Sara Kapasi, Christian M. Williams, Vannessa Doulames, Samya Sen, Manoj K. Manna, Alakesh Alakesh, Katie Lu, Ian Hall, Eric A. Appel

{"title":"Long-Acting Hydrogel-Based Depot Formulations of Tirzepatide and Semaglutide for the Management of Type 2 Diabetes and Weight","authors":"Andrea I. d'Aquino, Changxin Dong, Leslee T. Nguyen, Jerry Yan, Carolyn K. Jons, Olivia M. Saouaf, Ye Eun Song, Noah Eckman, Sara Kapasi, Christian M. Williams, Vannessa Doulames, Samya Sen, Manoj K. Manna, Alakesh Alakesh, Katie Lu, Ian Hall, Eric A. Appel","doi":"10.1002/adtp.202500329","DOIUrl":"10.1002/adtp.202500329","url":null,"abstract":"<div>\u0000 \u0000 <p>Several incretin hormone therapies have been clinically approved and have revolutionized the treatment of diabetes and obesity. Promising therapeutics include semaglutide (Ozempic and Wegovy), a glucagon-like peptide-1 (GLP-1) receptor agonist, and tirzepatide (Mounjaro), a dual agonist for GLP-1 and glucose-dependent insulinotropic polypeptide (GIP) receptors. These molecules help regulate blood glucose levels, enhance insulin secretion and sensitivity, and reduce appetite. Currently, these treatments require weekly injections, which can be challenging for patients to adhere to. We previously reported the development of an injectable hydrogel depot technology enabling months-long release of semaglutide (Sema). Here, we further developed this technology for improved prolonged release of both Sema and tirzepatide (TZP). In a type 2 diabetes rat model, we show that a single administration of hydrogel-based formulations of either Sema or TZP maintained relevant drug levels for over 6 weeks. In these studies, single administrations of long-acting hydrogel-based therapies of Sema or TZP were similarly effective at regulating blood glucose and weight compared to daily injections of either Sema or TZP in standard aqueous vehicles. This injectable hydrogel depot is easy to manufactureand exhibits excellent biocompatibility, enabling months-long treatments with the potential to improve the management of diabetes and weight.</p>\u0000 </div>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 2","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147288486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Silk Fibroin in Medicine: A Review on ISO10993 Biocompatibility, Preclinical Evidence, and Clinical Perspectives 丝素蛋白在医学上的应用：ISO10993生物相容性、临床前证据和临床前景综述

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-22 DOI: 10.1002/adtp.202500483

Jirasak Jitpibull, Juthamas Ratanavaraporn

{"title":"Silk Fibroin in Medicine: A Review on ISO10993 Biocompatibility, Preclinical Evidence, and Clinical Perspectives","authors":"Jirasak Jitpibull, Juthamas Ratanavaraporn","doi":"10.1002/adtp.202500483","DOIUrl":"10.1002/adtp.202500483","url":null,"abstract":"<div>\u0000 \u0000 <p>Silk fibroin, a natural protein polymer derived from silkworms, has been widely utilized in the development of biomaterials due to its numerous advantageous properties, including versatile processability into various formats, tunable scaffold characteristics, extended biodegradation time, and low immunogenicity in the human body. This review provides a comprehensive overview of the fundamental properties of silk fibroin—such as its thermal, mechanical, optical, and electrical characteristics—offering insights that can guide the tailoring of this material for specific biomedical applications. It also summarizes relevant safety data, including ISO 10993 series evaluations, which confirm the biocompatibility of silk fibroin by demonstrating its low toxicity, blood compatibility, mild inflammatory response, absence of skin sensitization, and lack of systemic toxicity. Furthermore, this review highlights findings from in vivo models across various tissue types, including skin, bone, and nerve, underscoring the high performance of silk fibroin-based materials in tissue repair and regeneration. Clinical trial reports are also discussed, demonstrating the therapeutic effectiveness, safety, and potential of silk fibroin-based materials for tissue repair and reconstruction in different organs, thereby reinforcing their translational promise for real-world medical applications.</p>\u0000 </div>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 2","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147288478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Radioisotope-Labeled Nanomedicines for Cancer Therapy: Recent Advances, Synergistic Strategies, and Future Challenges 放射性同位素标记的纳米药物用于癌症治疗：最新进展，协同策略和未来挑战

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-16 DOI: 10.1002/adtp.202500401

Zhisheng Luo, Lu Hao, Peng Liu, Shuo Hu

{"title":"Radioisotope-Labeled Nanomedicines for Cancer Therapy: Recent Advances, Synergistic Strategies, and Future Challenges","authors":"Zhisheng Luo, Lu Hao, Peng Liu, Shuo Hu","doi":"10.1002/adtp.202500401","DOIUrl":"10.1002/adtp.202500401","url":null,"abstract":"<div>\u0000 \u0000 <p>Radioisotope therapy (RIT), a form of internal radiotherapy, eliminates tumors at close range through the emission of α particles, β particles, or Auger electrons during radionuclide decay. It has been extensively applied in the treatment of advanced localized cancers. However, the precise delivery of radionuclides to tumor sites is essential to ensure both the safety and efficacy of RIT. Additionally, intrinsic or acquired tumor resistance can significantly compromise therapeutic outcomes. In recent decades, nanocarriers have been widely investigated for radioisotope delivery, enabling the development of diverse radionuclide-labeled nanomaterials and innovative cancer treatment strategies. This review summarizes therapeutic radioisotopes emitting α, β, or Auger particles and highlights various radionuclide-labeled nanomedicines, including inorganic nanoparticles, organic nanoparticles, and metal–organic frameworks (MOFs). We further discuss the integration of RIT with other modalities, such as chemotherapy, immunotherapy, hyperthermia, and photodynamic therapy, using nanomedicines. Finally, we outline the future prospects and challenges associated with the development and clinical translation of radionuclide-labeled nanomedicines.</p>\u0000 </div>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 2","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146217221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Front Cover: Zinc-Based Nanocomposites for Alzheimer's Therapy: Restoring Metal Homeostasis and Inhibiting Neurotoxic Protein Aggregation (Adv. Therap. 2/2026) 封面：锌基纳米复合材料用于阿尔茨海默病治疗：恢复金属稳态和抑制神经毒性蛋白聚集（ad . Therapy . 2/2026）

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-15 DOI: 10.1002/adtp.70133

Suman Yadav, Sarvesh Kumar Pandey, Shikha Awasthi

引用次数: 0

Antimalarial Polyethylene Glycol–Lumefantrine Conjugates: Synthesis and Effect of Linker Chemistry on Drug Release 抗疟聚乙二醇-甲苯胺缀合物的合成及其对药物释放的影响

IF 2.6 4区医学

Advanced Therapeutics Pub Date : 2026-02-12 DOI: 10.1002/adtp.202500418

Thenesia R. Govender, William M. R. Matshe, Nokuthula Dube, Zamani E. D. Cele, Aurelia A. Williams, Lynne A. Pilcher, Mohammed O. Balogun

{"title":"Antimalarial Polyethylene Glycol–Lumefantrine Conjugates: Synthesis and Effect of Linker Chemistry on Drug Release","authors":"Thenesia R. Govender, William M. R. Matshe, Nokuthula Dube, Zamani E. D. Cele, Aurelia A. Williams, Lynne A. Pilcher, Mohammed O. Balogun","doi":"10.1002/adtp.202500418","DOIUrl":"10.1002/adtp.202500418","url":null,"abstract":"<p>Polymer–drug conjugates have seen limited exploration in antimalarial therapy, despite their successful development for cancer and other diseases. With rising resistance and a critical shortage of first-line treatments for severe malaria, innovative drug delivery strategies are urgently needed to maximize the currently available drugs. Building on our previous work that demonstrated a water-soluble polymer–lumefantrine conjugate for the intravenous treatment of severe malaria, we investigated the influence of linker chemistry on drug release rate and kinetics of a new polyethylene glycol-lumefantrine conjugate under conditions relevant to malaria pathophysiology. Four homologous aliphatic diacid linkers (succinic, glutaric, adipic, and dodecanedioic acids) containing 4, 5, 6, and 12 carbon atoms, respectively, were introduced between the polymer and the drug. The conjugates were structurally well-defined and selectively cleaved at the ester bond under acidic conditions (pH 5.5), releasing only free lumefantrine, while remaining stable in human plasma (pH 7.4). Drug release rates were inversely proportional to linker length, with only the succinic acid-linked conjugate, which exhibited an initial burst release, deviating from first-order kinetic models. Complete inhibition of the <i>Plasmodium falciparum</i> NF54 strain was observed in vitro with a divalent variant of the succinic acid-linked conjugate, underscoring its potential for effective therapeutic action.</p>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"9 2","pages":""},"PeriodicalIF":2.6,"publicationDate":"2026-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://advanced.onlinelibrary.wiley.com/doi/epdf/10.1002/adtp.202500418","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146216957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0