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Bioorthogonal Engineered Virus‑Like Nanoparticles for Efficient Gene therapy

纳微快报:英文版 2023年第11期15卷 119-135页

作者： Chun‑Jie Bao Jia‑Lun Duan Ying Xie Xin‑Ping Feng Wei Cui Song‑Yue Chen Pei‑Shan Li Yi‑Xuan Liu Jin‑Ling Wang Gui‑Ling Wang Wan‑Liang Lu State Key Laboratory of Natural and Biomimetic Drugs Beijing Key Laboratory of Molecular Pharmaceutics and Drug Delivery Systemsand School of Pharmaceutical SciencesPeking UniversityBeijing 100191People’s Republic of China Jiangsu Collaborative Innovation Center of Chinese Medicinal Resources Industrialization Nanjing University of Chinese MedicineNanjing 210023People’s Republic of China School of Medicine&Holistic Integrative Medicine Nanjing University of Chinese MedicineNanjing 210023People’s Republic of China School of Chemical Sciences University of Chinese Academy of SciencesBeijing 100049People’s Republic of China

Gene therapy offers potentially transformative strategies for major human diseases.However,one of the key challenges in Gene therapy is developing an effective strategy that could deliver genes into the specific tissue.Here,we report a novel virus-like nanoparticle,the bioorthgonal engineered viruslike recombinant biosome(reBiosome),for efficient gene therapies of cancer and inflammatory diseases.The mutant virus-like biosome(mBiosome)is first prepared by site-specific codon mutation for displaying 4-azido-L-phenylalanine on vesicular stomatitis virus glycoprotein of eBiosome at a rational site,and the reBiosome is then prepared by clicking weak acid-responsive hydrophilic polymer onto the mBiosome via bioorthogonal chemistry.The results show that the reBiosome exhibits reduced virus-like immunogenicity,prolonged blood circulation time and enhanced gene delivery efficiency to weakly acidic foci(like tumor and arthritic tissue).Furthermore,reBiosome demonstrates robust therapeutic efficacy in breast cancer and arthritis by delivering gene editing and silencing systems,respectively.In conclusion,this study develops a universal,safe and efficient platform for gene therapies for cancer and inflammatory diseases.

关键词： Virus-like nanoparticle Site-specific codon mutation Recombinant biosome Bioorthogonal chemistry Gene therapy

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CMT1A current Gene therapy approaches and promising biomarkers

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Neural Regeneration Research 2023年第7期18卷 1434-1440页

作者： Marina Stavrou Kleopas AKleopa Neuroscience Department The Cyprus Institute of Neurology and GeneticsNicosiaCyprus Center for Neuromuscular Disorders The Cyprus Institute of Neurology and GeneticsNicosiaCyprus

Charcot-Marie-Tooth neuropathies(CMT)constitute a group of common but highly heterogeneous,non-syndromic genetic disorders affecting predominantly the peripheral nervous system.CMT type 1A(CMT1A)is the most frequent type and accounts for almost~50%of all diagnosed CMT cases.CMT1A results from the duplication of the peripheral myelin protein 22(PMP22)gene.Overexpression of PMP22 protein overloads the protein folding apparatus in Schwann cells and activates the unfolded protein response.This leads to Schwann cell apoptosis,dys-and de-myelination and secondary axonal degeneration,ultimately causing neurological disabilities.During the last decades,several different gene therapies have been developed to treat CMT1A.Almost all of them remain at the pre-clinical stage using CMT1A animal models overexpressing PMP22.The therapeutic goal is to achieve gene silencing,directly or indirectly,thereby reversing the CMT1A genetic mechanism allowing the recovery of myelination and prevention of axonal loss.As promising treatments are rapidly emerging,treatment-responsive and clinically relevant biomarkers are becoming necessary.These biomarkers and sensitive clinical evaluation tools will facilitate the design and successful completion of future clinical trials for CMT1A.

关键词： axonal degeneration biomarkers Charcot-Marie-Tooth disease Gene therapy inherited neuropathy mouse models

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Ionizable drug delivery systems for efficient and selective Gene therapy

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Military Medical Research 2023年第6期10卷 818-847页

作者： Yu-Qi Zhang Ran-Ran Guo Yong-Hu Chen Tian-Cheng Li Wen-Zhen Du Rong-Wu Xiang Ji-Bin Guan Yu-Peng Li Yuan-Yu Huang Zhi-Qiang Yu Yin Cai Peng Zhang Gui-Xia Ling Faculty of Medical Device Shenyang Pharmaceutical UniversityShenyang 110016LiaoningChina Wuya College of Innovation Shenyang Pharmaceutical UniversityShenyang 110016LiaoningChina School of Pharmacy Yanbian UniversityYanji 133002JilinChina School of Pharmacy Shenyang Pharmaceutical UniversityShenyang 110016LiaoningChina Masonic Cancer Center University of MinnesotaMinneapolisMN 55455USA Masonic Cancer Center and Department of Medicinal Chemistry University of MinnesotaMinneapolisMN 55455USA Advanced Research Institute of Multidisciplinary Science School of Life ScienceSchool of Medical TechnologyKey Laboratory of Molecular Medicine and BiotherapyKey Laboratory of Medical Molecule Science and Pharmaceutics EngineeringBeijing Institute of TechnologyBeijing 100081China Department of Laboratory Medicine Dongguan Institute of Clinical Cancer ResearchAffiliated Dongguan HospitalSouthern Medical UniversityDongguan 523018GuangdongChina Department of Health Technology and Informatics The Hong Kong Polytechnic UniversityHong KongHong Kong SARChina

Gene therapy has shown great potential to treat various diseases by repairing the abnormal gene function.However,a great challenge in bringing the nucleic acid formulations to the market is the safe and effective delivery to the specific tissues and cells.To be excited,the development of ionizable drug delivery systems(IDDSs)has promoted a great breakthrough as evidenced by the approval of the BNT162b2 vaccine for prevention of coronavirus disease 2019(COVID-19)in 2021.Compared with conventional cationic gene vectors,IDDSs can decrease the toxicity of carriers to cell membranes,and increase cellular uptake and endosomal escape of nucleic acids by their unique pH-responsive structures.Despite the progress,there remain necessary requirements for designing more efficient IDDSs for precise Gene therapy.Herein,we systematically classify the IDDSs and summarize the characteristics and advantages of IDDSs in order to explore the underlying design mechanisms.The delivery mechanisms and therapeutic applications of IDDSs are comprehensively reviewed for the delivery of plasmid DNA(pDNA)and four kinds of RNA.In particular,organ selecting considerations and high-throughput screening are highlighted to explore efficiently multifunctional ionizable nanomaterials with superior gene delivery capacity.We anticipate providing references for researchers to rationally design more efficient and accurate targeted gene delivery systems in the future,and indicate ideas for developing next generation gene vectors.

关键词： Ionizable nanomaterials Ionizable drug delivery systems(IDDSs) Nucleic acids Gene therapy

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Nanotechnology-based Gene therapy as a credible tool in the treatment of Alzheimer’s disease

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Neural Regeneration Research 2023年第10期18卷 2127-2133页

作者： Aziz Unnisa Nigel H.Greig Mohammad Amjad Kamal Department of Pharmaceutical Chemistry College of PharmacyUniversity of HailHailSaudi Arabia Drug Design&Development Section Translational Gerontology BranchIntramural Research ProgramNational Institute on AgingNational Institutes of HealthBaltimoreMDUSA Institutes for Systems genetics Frontiers Science Center for Disease-related Molecular NetworkWest China HospitalSichuan UniversityChengduSichuan ProvinceChina King Fahd Medical Research Center King Abdulaziz UniversityJeddahSaudi Arabia Department of Pharmacy Faculty of Allied Health SciencesDaffodil International UniversityDhakaBangladesh Enzymoics Novel Global Community Educational FoundationHebershamNSWAustralia

Toxic aggregated amyloid-βaccumulation is a key pathogenic event in Alzheimer’s disease.Treatment approaches have focused on the suppression,deferral,or dispersion of amyloid-βfibers and plaques.Gene therapy has evolved as a potential therapeutic option for treating Alzheimer’s disease,owing to its rapid advancement over the recent decade.Small interfering ribonucleic acid has recently garnered considerable attention in Gene therapy owing to its ability to down-regulate genes with high sequence specificity and an almost limitless number of therapeutic targets,including those that were once considered undruggable.However,lackluster cellular uptake and the destabilization of small interfering ribonucleic acid in its biological environment restrict its therapeutic application,necessitating the development of a vector that can safeguard the genetic material from early destruction within the bloodstream while effectively delivering therapeutic genes across the bloodbrain barrier.Nanotechnology has emerged as a possible solution,and several delivery systems utilizing nanoparticles have been shown to bypass key challenges regarding small interfering ribonucleic acid delivery.By reducing the enzymatic breakdown of genetic components,nanomaterials as gene carriers have considerably enhanced the efficiency of Gene therapy.Liposomes,polymeric nanoparticles,magnetic nanoparticles,dendrimers,and micelles are examples of nanocarriers that have been designed,and each has its own set of features.Furthermore,recent advances in the specific delivery of neurotrophic compounds via Gene therapy have provided promising results in relation to augmenting cognitive abilities.In this paper,we highlight the use of different nanocarriers in targeted gene delivery and small interfering ribonucleic acid-mediated gene silencing as a potential platform for treating Alzheimer’s disease.

关键词： Alzheimer’s disease amyloid-β BACE1 gene silencing Gene therapy nanoparticle neurotrophins small interfering ribonucleic acid

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Brain and spinal cord trauma:what we know about the therapeutic potential of insulin growth factor 1 Gene therapy

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Neural Regeneration Research 2023年第2期18卷 253-257页

作者： María Jose Bellini Florencia Labombarda Lab de Bioquímica del Envejecimiento INIBIOLP-CONICETFacultad de Ciencias MedicinaUniversidad Nacional de La PlataLa PlataArgentina Laboratory of Neuroendocrine Biochemistry IBYME-CONICETBuenos AiresArgentina Department of Human Biochemistry Faculty of MedicineUniversidad de Buenos AiresBuenos AiresArgentina

Although little attention has been paid to cognitive and emotional dysfunctions observed in patients after spinal co rd injury,several reports have described impairments in cognitive abilities.Our group also has contributed significantly to the study of cognitive impairments in a rat model of spinal co rd injury.These findings are very significant because they demonstrate that cognitive and mood deficits are not induced by lifestyle changes,drugs of abuse,and combined medication.They are related to changes in brain structures involved in cognition and emotion,such as the hippocampus.Chronic spinal cord injury decreases neurogenesis,enhances glial reactivity leading to hippocampal neuroinflammation,and trigge rs cognitive deficits.These brain distal abnormalities are recently called te rtiary damage.Given that there is no treatment for Tertiary Damage,insulin growth factor 1 Gene therapy emerges as a good candidate.Insulin growth factor 1 gene thera py recove rs neurogenesis and induces the polarization from pro-inflammato ry towards anti-inflammatory microglial phenotypes,which represents a potential strategy to treat the neuroinflammation that supports te rtiary damage.Insulin growth factor 1 Gene therapy can be extended to other central nervous system pathologies such as traumatic brain injury where the neuroinflammatory component is crucial.Insulin growth factor 1 Gene therapy could emerge as a new therapeutic strategy for treating traumatic brain injury and spinal cord injury.

关键词： cognitive impairments Gene therapy hippocampus insulin growth factor 1 microglial cells neurodegeneration neurogenesis neuroinflammation spinal cord injury traumatic brain injury

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Gene therapy Activates Retinal Pigment Epithelium Cell Proliferation for Age-related Macular Degeneration in a Mouse Model

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Current Medical Science 2023年第2期43卷 384-392页

作者： Yun YUAN Wen KONG Xiao-mei LIU Guo-hua SHI School of Biomedical Engineering(Suzhou) Division of Life Sciences and MedicineUniversity of Science and Technology of ChinaSuzhou215000China Suzhou Institute of Biomedical Engineering and Technology Chinese Academy of ScienceSuzhou215000China

Objective Age-related macular degeneration(AMD)is a degenerative retinal disease.The degeneration or death of retinal pigment epithelium(RPE)cells is implicated in the pathogenesis of AMD.This study aimed to activate the proliferation of RPE cells in vivo by using an adeno-associated virus(AAV)vector encodingβ-catenin to treat AMD in a mouse model.Methods Mice were intravitreally injected with AAV2/8-Y733F-VMD2-β-catenin for 2 or 4 weeks,andβ-catenin expression was measured using immunofluorescence staining,real-time quantitative reverse transcription polymerase chain reaction(PCR),and Western blotting.The function ofβ-catenin was determined using retinal flat mounts and laser-induced damage models.Finally,the safety of AAV2/8-Y733F-VMD2-β-catenin was evaluated by multiple intravitreal injections.Results AAV2/8-Y733F-VMD2-β-catenin induced the expression ofβ-catenin in RPE cells.It activated the proliferation of RPE cells and increased cyclin D1 expression.It was beneficial to the recovery of laser-induced damage by activating the proliferation of RPE cells.Furthermore,it could induce apoptosis of RPE cells by increasing the expression of Trp53,Bax and caspase3 while decreasing the expression of Bcl-2.Conclusion AAV2/8-Y733F-VMD2-β-catenin increasedβ-catenin expression in RPE cells,activated RPE cell proliferation,and helped mice heal from laser-induced eye injury.Furthermore,it could induce the apoptosis of RPE cells.Therefore,it may be a safe approach for AMD treatment.

关键词： Gene therapy adeno-associated virus age-related macular degeneration retinal pigment epithelium cells β-catenin

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Metformin booster adipocyte-targeted Gene therapy for the treatment of obesity and related metabolic syndromes

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Science China Chemistry 2022年第4期65卷 796-809页

作者： Jie Chen Jee Young Chung Huapan Fang Lin Lin Yong-Hee Kim Huayu Tian Xuesi Chen Key Laboratory of Polymer Ecomaterials Changchun Institute of Applied ChemistryChinese Academy of SciencesChangchun 130022China Department of Bioengineering Institute for Bioengineering and Biopharmaceutical ResearchBK 21 Plus Future Biopharmaceutical Human Resources Training and Research TeamHanyang University133-791 SeoulRepublic of Korea

Obesity has become an important public problem that endangers human conditions and urgently needs to be solved. However,most weight-loss drugs on the market have little effect and are accompanied by adverse effects such as strokes and heart attacks.Here, we construct an adipocyte-targeting polypeptide-based gene carrier consisting of an adipocyte-targeting peptide and ptoluylsulfonyl arginine-modified polylysine(ATS-PLL-RT), which can specifically bind to the prohibitin of mature adipocytes.We further construct a short hairpin RNA(shRNA) to simultaneously silence fatty acid binding proteins 4 and 5(shFABP4/5).FABPs are molecular chaperones for fatty acid metabolism and storage in cells. Moreover, we introduce metformin for combined therapy. First, the metformin combination can effectively improve the efficiency of gene transfection. In addition, metformin itself has an alleviating effect on diet-induced obesity and relevant metabolic diseases. The combination treatment of obese mice with ATS-PLL-RT/shFABP4/5 and metformin achieves body weight reduction and metabolic recovery. This study provides a potentially effective strategy for the clinical treatment of obesity as well as mitigating obesity-induced metabolic syndromes.

关键词： FABP4/5 Gene therapy insulin resistance metformin obesity

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Application of Gene therapy in Hemophilia

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Current Medical Science 2022年第5期42卷 925-931页

作者： Yue-fen HU Yun-hai FANG Yong-rong LAI Xiao-qin FENG Shu-qian XU Department of Hematology Qilu HospitalCheeloo College of MedicineShandong UniversityJinan 250014China Shandong Blood Center Shandong Hemophilia Treatment CenterJinan 250014China Department of Hematology the First Affiliated Hospital of Guangxi Medical UniversityNanning 530021China Nanfang Hospital Southern Medical UniversityGuangzhou 510000China

Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and abnormal genes for the purpose of therapy.It holds out hope of a cure for single-gene genetic diseases such as thalassemia,hemophilia,etc.At present,Gene therapy is performed in two ways:introducing exogenous genes,and gene editing.A great number of clinical trials of Gene therapy in hemophilia have been carried out using viral vectors to introduce foreign genes into target cells.However,the production of neutralizing antibodies following injection and the inability to prepare viral vectors in large quantities limit their application.Although gene-editing methods like CRISPR avoid the above problems,the potential risks of off-target effects are still unknown.More trials and evidence are needed to elucidate the safety and accuracy of Gene therapy.This paper will review the bench and clinical work of Gene therapy in hemophilia in recent years,and summarize the challenges and prospects of Gene therapy,so as to provide directions for future scientific research in this field.

关键词： hemophilia Gene therapy CRISPR-Cas9 application

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Biomaterial-assisted biotherapy:A brief review of biomaterials used in drug delivery,vaccine development,Gene therapy,and stem cell therapy

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Bioactive Materials 2022年第11期7卷 29-48页

作者： Xuejiao Han Aqu Alu Hongmei Liu Yi Shi Xiawei Wei Lulu Cai Yuquan Wei Laboratory of Aging Research and Cancer Drug Target State Key Laboratory of BiotherapyNational Clinical Research Center for GeriatricsWest China HospitalSichuan UniversityChengduChina Personalized Drug therapy Key Laboratory of Sichuan Province Department of PharmacySichuan Provincial People’s HospitalSchool of MedicineUniversity of Electronic Science and Technology of ChinaChengdu610072China Sichuan Provincial Key Laboratory for Human Disease gene Study and Department of Laboratory Medicine Sichuan Provincial People’s HospitalUniversity of Electronic Science and Technology of ChinaChengdu610072China

Biotherapy has recently become a hotspot research topic with encouraging prospects in various fields due to a wide range of treatments applications,as demonstrated in preclinical and clinical studies.However,the broad applications of biotherapy have been limited by critical challenges,including the lack of safe and efficient de-livery systems and serious side effects.Due to the unique potentials of biomaterials,such as good biocompati-bility and bioactive properties,biomaterial-assisted biotherapy has been demonstrated to be an attractive strategy.The biomaterial-based delivery systems possess sufficient packaging capacity and versatile functions,enabling a sustained and localized release of drugs at the target sites.Furthermore,the biomaterials can provide a niche with specific extracellular conditions for the proliferation,differentiation,attachment,and migration of stem cells,leading to tissue regeneration.In this review,the state-of-the-art studies on the applications of bio-materials in biotherapy,including drug delivery,vaccine development,Gene therapy,and stem cell therapy,have been summarized.The challenges and an outlook of biomaterial-assisted biotherapies have also been discussed.

关键词： Biomaterial Biotherapy Drug delivery system Vaccine development Gene therapy Stem cell therapy

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A non-viral Gene therapy for melanoma by staphylococcal enterotoxin A

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Chinese Chemical Letters 2024年第5期35卷 325-329页

作者： Ling Yang Min Ren Jie Wang Liming He Shanshan Wu Shuai Yang Wei Zhao Hao Cheng Xiaoming Zhou Maling Gou State Key Laboratory of Biotherapy and Cancer Center West China HospitalSichuan UniversityChengdu 610041China Department of Ophthalmology West China HospitalSichuan UniversityChengdu 610041China State Key Laboratory of Biotherapy Department of Integrated Traditional Chinese and Western MedicineRare Diseases CenterWest China HospitalSichuan UniversityChengdu 610041China

Staphylococcal enterotoxin A(SEA)derived from Staphylococcus aureus,as a superantigen,shows potential for cancer immunotherapy,but systemic immunotoxicity restricts its clinical application.Targeted delivery of SEA to tumor site provides a promising option for reducing the systemic toxicity.Here,we constructed an iRGD peptide(H-[Cys-Arg-Gly-Asp-Lys-Gly-Pro-Asp-Cys]-NH_(2))modified nanoparticle(iDPP)to deliver plasmids encoding SEA for melanoma treatment.The iDPP/SEA nanocomplexes efficiently mediated SEA expression in B16-F10 cells in vivo and in vitro and induced the activation of lymphocytes and maturation of murine bone marrow-derived dendritic cells(BMDCs)in vitro.In the subcutaneous B16-F10 melanoma model,the iDPP/SEA nanocomplexes could effectively enhance immune response and T lymphocytes infiltration in tumor site after intravenous administration,thereby considerably decreased melanoma growth.Meanwhile,no obvious adverse effect was observed after intravenous administration of the iDPP/SEA nanocomplexes in vivo.Our findings demonstrated that Gene therapy of SEA is a potential candidate for melanoma treatment.

关键词： Gene therapy Superantigen Melanoma Staphylococcal enterotoxins A Immunotherapyene therapy

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