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Page 1
Gene therapy in cancer.
Cesur-Ergün B, Demir-Dora D. Cesur-Ergün B, et al. J Gene Med. 2023 Nov;25(11):e3550. doi: 10.1002/jgm.3550. Epub 2023 Jun 24. J Gene Med. 2023. PMID: 37354071 Review.
This treatment has various strategies such as therapeutic gene activation or silencing of unwanted or defective genes; therefore a wide variety of genes and viral or nonviral vectors are being used in studies. Gene therapy strategies in cancer can be classified as i …
This treatment has various strategies such as therapeutic gene activation or silencing of unwanted or defective genes; therefore a wide vari …
Gene therapy: a double-edged sword with great powers.
Tang R, Xu Z. Tang R, et al. Mol Cell Biochem. 2020 Nov;474(1-2):73-81. doi: 10.1007/s11010-020-03834-3. Epub 2020 Jul 21. Mol Cell Biochem. 2020. PMID: 32696132 Review.
However, concerns about its efficacy and safety have accompanied gene therapy since its birth. In the present manuscript, we first introduce various strategies employed in gene therapy, which includes ex vivo gene delivery v.s. in vivo gene delivery; g …
However, concerns about its efficacy and safety have accompanied gene therapy since its birth. In the present manuscript, we f …
Gene therapy.
Brody H. Brody H. Nature. 2018 Dec;564(7735):S5. doi: 10.1038/d41586-018-07639-9. Nature. 2018. PMID: 30542191 No abstract available.
Various AAV Serotypes and Their Applications in Gene Therapy: An Overview.
Issa SS, Shaimardanova AA, Solovyeva VV, Rizvanov AA. Issa SS, et al. Cells. 2023 Mar 1;12(5):785. doi: 10.3390/cells12050785. Cells. 2023. PMID: 36899921 Free PMC article. Review.
Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Today, many AAV-based gene therapy medications are being investigated in preclinical a …
Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases ba …
Applications of Gene Therapy in Cardiomyopathies.
Argiro A, Bui Q, Hong KN, Ammirati E, Olivotto I, Adler E. Argiro A, et al. JACC Heart Fail. 2024 Feb;12(2):248-260. doi: 10.1016/j.jchf.2023.09.015. Epub 2023 Oct 7. JACC Heart Fail. 2024. PMID: 37966402 Free article. Review.
Notably, gene therapy for Duchenne muscular dystrophy has recently been approved and pivotal clinical trials are testing gene therapy approaches in rare myocardial conditions such as Danon disease and Fabry disease. Furthermore, promising results have …
Notably, gene therapy for Duchenne muscular dystrophy has recently been approved and pivotal clinical trials are testing ge …
Advances in gene therapy hold promise for treating hereditary hearing loss.
Jiang L, Wang D, He Y, Shu Y. Jiang L, et al. Mol Ther. 2023 Apr 5;31(4):934-950. doi: 10.1016/j.ymthe.2023.02.001. Epub 2023 Feb 8. Mol Ther. 2023. PMID: 36755494 Free PMC article. Review.
Therefore, in order to further advance the development of inner ear gene therapy and promote its broad application in other forms of genetic disease, it is imperative to review the progress of gene therapy for HHL. ...Finally, we elaborate on the chall …
Therefore, in order to further advance the development of inner ear gene therapy and promote its broad application in other fo …
Gene therapy for heart failure and cardiomyopathies.
Argirò A, Ding J, Adler E. Argirò A, et al. Rev Esp Cardiol (Engl Ed). 2023 Dec;76(12):1042-1054. doi: 10.1016/j.rec.2023.06.009. Epub 2023 Jul 26. Rev Esp Cardiol (Engl Ed). 2023. PMID: 37506969 Free article. Review. English, Spanish.
Gene therapy strategies encompass a range of approaches, including gene replacement and gene editing. ...This review aims to summarize the mechanisms behind different gene therapy strategies, the available delivery systems, the primary risks associated …
Gene therapy strategies encompass a range of approaches, including gene replacement and gene editing. ...This review aims to s …
Gene therapy clinical trials, where do we go? An overview.
Arabi F, Mansouri V, Ahmadbeigi N. Arabi F, et al. Biomed Pharmacother. 2022 Sep;153:113324. doi: 10.1016/j.biopha.2022.113324. Epub 2022 Jun 29. Biomed Pharmacother. 2022. PMID: 35779421 Free article. Review.
There have been many ups and downs since the introduction of gene therapy as a therapeutic modality for diseases. However, the journey of gene therapy has reached a fundamental milestone, as evidenced by the increasing number of gene therapy …
There have been many ups and downs since the introduction of gene therapy as a therapeutic modality for diseases. However, the …
Advances in HIV Gene Therapy.
Kitawi R, Ledger S, Kelleher AD, Ahlenstiel CL. Kitawi R, et al. Int J Mol Sci. 2024 Feb 28;25(5):2771. doi: 10.3390/ijms25052771. Int J Mol Sci. 2024. PMID: 38474018 Free PMC article. Review.
Recent advances in genetic engineering technologies have reignited interest, leading to the approval of the first gene therapy product targeting genetic mutations in 2017. Gene therapy (GT) can be delivered either in vivo or ex vivo. ...This review hig …
Recent advances in genetic engineering technologies have reignited interest, leading to the approval of the first gene therapy …
Ewing sarcoma treatment: a gene therapy approach.
Tsibulnikov S, Fayzullina D, Karlina I, Schroeder BA, Karpova O, Timashev P, Ulasov I. Tsibulnikov S, et al. Cancer Gene Ther. 2023 Aug;30(8):1066-1071. doi: 10.1038/s41417-023-00615-0. Epub 2023 Apr 10. Cancer Gene Ther. 2023. PMID: 37037906 Free PMC article. Review.
Fusion genes and fusion protein products are promising targets for gene therapy. Therapeutic approaches and strategies vary based on target molecules (nucleotides, proteins) of interest. We present an extensive literature review of active molecules for gene …
Fusion genes and fusion protein products are promising targets for gene therapy. Therapeutic approaches and strategies vary ba …
CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future.
Uddin F, Rudin CM, Sen T. Uddin F, et al. Front Oncol. 2020 Aug 7;10:1387. doi: 10.3389/fonc.2020.01387. eCollection 2020. Front Oncol. 2020. PMID: 32850447 Free PMC article. Review.
The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) proteins has expanded the applications of genetic research in thousands of laboratories across the globe and is redefining our approach to gene therapy. T …
The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) proteins has expanded the ap …
Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues.
Leebeek FWG, Miesbach W. Leebeek FWG, et al. Blood. 2021 Sep 16;138(11):923-931. doi: 10.1182/blood.2019003777. Blood. 2021. PMID: 34232980 Free article. Review.
In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered adeno-associated virus (AAV)-based liver-directed gene therapy in patients …
In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first …
Current State of Human Gene Therapy: Approved Products and Vectors.
Shchaslyvyi AY, Antonenko SV, Tesliuk MG, Telegeev GD. Shchaslyvyi AY, et al. Pharmaceuticals (Basel). 2023 Oct 5;16(10):1416. doi: 10.3390/ph16101416. Pharmaceuticals (Basel). 2023. PMID: 37895887 Free PMC article. Review.
In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking identification of the first recombinant DNA in 1972. ...The article also discusses the advantages and disadvantages of each product and vector type, as well as the current challen …
In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking identification of the first recombinant …
Liver Gene Therapy.
Nathwani AC, McIntosh J, Sheridan R. Nathwani AC, et al. Hum Gene Ther. 2022 Sep;33(17-18):879-888. doi: 10.1089/hum.2022.169. Hum Gene Ther. 2022. PMID: 36082993 Review.
Gene therapy is an exciting therapeutic concept that offers the promise of a cure for an array of inherited and acquired disorders. The liver has always been a key target for gene therapy as it controls essential biological processes including digestio …
Gene therapy is an exciting therapeutic concept that offers the promise of a cure for an array of inherited and acquired disor …
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond.
Duan D. Duan D. Hum Gene Ther. 2023 May;34(9-10):345-349. doi: 10.1089/hum.2023.29242.ddu. Hum Gene Ther. 2023. PMID: 37219994 Free PMC article.
In this issue of Human Gene Therapy, several research articles highlighted some of the latest developments in DMD gene therapy. Importantly, a collection of articles from experts in the field reviewed the progress, major challenges, and future directio …
In this issue of Human Gene Therapy, several research articles highlighted some of the latest developments in DMD gene …
Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review.
Ay C, Frenzel L, Pinachyan K, Le Quellec S. Ay C, et al. Haemophilia. 2024 Jan;30(1):5-15. doi: 10.1111/hae.14907. Epub 2023 Dec 18. Haemophilia. 2024. PMID: 38111029 Review.
AIM: To provide a comprehensive summary of the whole process of AAV-based gene therapy from basic principles to clinical implementation through an illustrated review. METHODS: The authors, with expertise in and knowledge about gene therapy for haemophi …
AIM: To provide a comprehensive summary of the whole process of AAV-based gene therapy from basic principles to clinical imple …
Fetal gene therapy.
Waddington SN, Peranteau WH, Rahim AA, Boyle AK, Kurian MA, Gissen P, Chan JKY, David AL. Waddington SN, et al. J Inherit Metab Dis. 2024 Jan;47(1):192-210. doi: 10.1002/jimd.12659. Epub 2023 Aug 7. J Inherit Metab Dis. 2024. PMID: 37470194 Free PMC article. Review.
Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene therapy was, to quote the Gartner hype cycle, at its "peak of inflated expectations." ...A confluence of clinical trials now signposts a trajectory toward fetal …
Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene therapy was, to quote t …
Prenatal Gene Therapy.
Schwab ME, MacKenzie TC. Schwab ME, et al. Clin Obstet Gynecol. 2021 Dec 1;64(4):876-885. doi: 10.1097/GRF.0000000000000655. Clin Obstet Gynecol. 2021. PMID: 34618719
Prenatal gene therapy could provide a cure for many monogenic diseases. Prenatal gene therapy has multiple potential advantages over postnatal therapy, including treating before the onset of disease, the ability to induce tolerance and cross the blood- …
Prenatal gene therapy could provide a cure for many monogenic diseases. Prenatal gene therapy has multiple poten …
Gene therapy for SCID, now up to 3!
Fischer A, Neven B. Fischer A, et al. J Allergy Clin Immunol. 2023 May;151(5):1255-1256. doi: 10.1016/j.jaci.2023.02.015. Epub 2023 Feb 23. J Allergy Clin Immunol. 2023. PMID: 36828079 No abstract available.
Gene therapy in epilepsy.
Zhang L, Wang Y. Zhang L, et al. Biomed Pharmacother. 2021 Nov;143:112075. doi: 10.1016/j.biopha.2021.112075. Epub 2021 Sep 3. Biomed Pharmacother. 2021. PMID: 34488082 Free article. Review.
Gene therapy may constitute a promising alternative to conventional pharmacological tools and surgeries for epilepsy. ...Creating gene vectors tailored for different situations is the key to expanding gene therapy, and choosing the appropriate therapeu …
Gene therapy may constitute a promising alternative to conventional pharmacological tools and surgeries for epilepsy. ...Creat …
Gene therapy vector-related myocarditis.
Silver E, Argiro A, Hong K, Adler E. Silver E, et al. Int J Cardiol. 2024 Mar 1;398:131617. doi: 10.1016/j.ijcard.2023.131617. Epub 2023 Nov 27. Int J Cardiol. 2024. PMID: 38030043 Free article. Review.
Gene therapy is a technique to correct genetic abnormalities, through introduction of a functional gene or through direct genome editing. ...This review explores the immunological reactions to AAV-based gene therapy, their potential toxic effects, with …
Gene therapy is a technique to correct genetic abnormalities, through introduction of a functional gene or through direct geno …
Cochlear gene therapy.
Lustig LR, Akil O. Lustig LR, et al. Curr Opin Neurol. 2012 Feb;25(1):57-60. doi: 10.1097/WCO.0b013e32834f038c. Curr Opin Neurol. 2012. PMID: 22157110 Free PMC article. Review.
PURPOSE OF REVIEW: This review highlights recent advances in cochlear gene therapy over the past several years. Cochlear gene therapy has undergone tremendous advances over the past decade. ...SUMMARY: If successful, cochlear gene therapy …
PURPOSE OF REVIEW: This review highlights recent advances in cochlear gene therapy over the past several years. Cochlear ge …
Gene therapy.
Baum BJ. Baum BJ. Oral Dis. 2014 Mar;20(2):115-8. doi: 10.1111/odi.12211. Epub 2013 Dec 26. Oral Dis. 2014. PMID: 24372817 Free PMC article.
Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should …
Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at l …
Human Cardiac Gene Therapy.
Ishikawa K, Weber T, Hajjar RJ. Ishikawa K, et al. Circ Res. 2018 Aug 17;123(5):601-613. doi: 10.1161/CIRCRESAHA.118.311587. Circ Res. 2018. PMID: 30355138 Free PMC article. Review.
Myocardial gene therapy has been tested in patients with heart failure using adeno-associated vectors with no safety concerns but lacking clinical improvements. Cardiac gene therapy is adapting to the new developments in vectors, delivery systems, targ …
Myocardial gene therapy has been tested in patients with heart failure using adeno-associated vectors with no safety concerns …
Gene therapy for retinitis pigmentosa.
Piri N, Grodsky JD, Kaplan HJ. Piri N, et al. Taiwan J Ophthalmol. 2021 Nov 19;11(4):348-351. doi: 10.4103/tjo.tjo_47_21. eCollection 2021 Oct-Dec. Taiwan J Ophthalmol. 2021. PMID: 35070662 Free PMC article. Review.
There is no proven cure for the disease, and multiple approaches are being studied. Gene therapy is an evolving field in medicine and ophthalmology. In this review, we will go over the basic concept of gene therapy and the different types of gene …
There is no proven cure for the disease, and multiple approaches are being studied. Gene therapy is an evolving field in medic …
Gene therapy research in Asia.
Deng HX, Wang Y, Ding QR, Li DL, Wei YQ. Deng HX, et al. Gene Ther. 2017 Sep;24(9):572-577. doi: 10.1038/gt.2017.62. Epub 2017 Sep 7. Gene Ther. 2017. PMID: 28726798 Review.
The clinical transformation and industrialization of gene therapy in Asia have been remarkable and continue making steady progress. A total of six gene therapy-based products have been approved worldwide, including two drugs from Asia. This review aims …
The clinical transformation and industrialization of gene therapy in Asia have been remarkable and continue making steady prog …
Gene Therapy in Heart Failure.
Fargnoli AS, Katz MG, Bridges CR, Hajjar RJ. Fargnoli AS, et al. Handb Exp Pharmacol. 2017;243:395-421. doi: 10.1007/164_2016_81. Handb Exp Pharmacol. 2017. PMID: 27796512 Review.
This chapter summarizes the key developments in cardiac gene therapy from the perspective of understanding each of these components of the treatment plan. The latest pharmacologic gene targets, gene therapy vectors, delivery routes, and strategies are …
This chapter summarizes the key developments in cardiac gene therapy from the perspective of understanding each of these compo …
Gene therapy for haemophilia.
Sharma A, Easow Mathew M, Sriganesh V, Reiss UM. Sharma A, et al. Cochrane Database Syst Rev. 2016 Dec 20;12(12):CD010822. doi: 10.1002/14651858.CD010822.pub3. Cochrane Database Syst Rev. 2016. Update in: Cochrane Database Syst Rev. 2020 Apr 28;4:CD010822. doi: 10.1002/14651858.CD010822.pub4. PMID: 27996087 Free PMC article. Updated.
DATA COLLECTION AND ANALYSIS: No trials of gene therapy for haemophilia were found. MAIN RESULTS: No trials of gene therapy for haemophilia were identified. AUTHORS' CONCLUSIONS: No randomised or quasi-randomised clinical trials of gene thera …
DATA COLLECTION AND ANALYSIS: No trials of gene therapy for haemophilia were found. MAIN RESULTS: No trials of gene …
Gene therapy review.
Moss JA. Moss JA. Radiol Technol. 2014 Nov-Dec;86(2):155-80; quiz 181-4. Radiol Technol. 2014. PMID: 25391667 Review.
Gene therapy holds the potential to control a wide range of diseases, including cystic fibrosis, heart disease, diabetes, cancer, and blood diseases. This review assesses the current status of gene therapy, highlighting therapeutic methodologies and ap …
Gene therapy holds the potential to control a wide range of diseases, including cystic fibrosis, heart disease, diabetes, canc …
Gene Therapy for Inherited Bleeding Disorders.
Arruda VR, Weber J, Samelson-Jones BJ. Arruda VR, et al. Semin Thromb Hemost. 2021 Mar;47(2):161-173. doi: 10.1055/s-0041-1722862. Epub 2021 Feb 26. Semin Thromb Hemost. 2021. PMID: 33636747 Free PMC article. Review.
Decades of preclinical and clinical studies developing gene therapy for hemophilia are poised to bear fruit with current promising pivotal studies likely to lead to regulatory approval. ...Preclinical studies have also investigated gene transfer approaches for other …
Decades of preclinical and clinical studies developing gene therapy for hemophilia are poised to bear fruit with current promi …
Gene therapy for inherited metabolic diseases.
Yilmaz BS, Gurung S, Perocheau D, Counsell J, Baruteau J. Yilmaz BS, et al. J Mother Child. 2020 Nov 10;24(2):53-64. doi: 10.34763/jmotherandchild.20202402si.2004.000009. J Mother Child. 2020. PMID: 33554501 Free PMC article. Review.
Over the last two decades, gene therapy has been successfully translated to many rare diseases. The number of clinical trials is rapidly expanding and some gene therapy products have now received market authorisation in the western world. ...We discuss …
Over the last two decades, gene therapy has been successfully translated to many rare diseases. The number of clinical trials …
From Mendel to Gene Therapy.
Jeffrey SL, Brigham DA, Chawla SP, Federman N, Hall FL, Gordon EM. Jeffrey SL, et al. Anticancer Res. 2023 Oct;43(10):4257-4261. doi: 10.21873/anticanres.16620. Anticancer Res. 2023. PMID: 37772566 No abstract available.
In Situ Gene Therapy.
Santos HS, Rodrigues L, Vera LNP, Poletto E, Filippi-Chiela E, Dos Santos Bruschi LFR, Schuh R, Baldo G. Santos HS, et al. Curr Gene Ther. 2021;21(5):406-430. doi: 10.2174/1566523221666210504103323. Curr Gene Ther. 2021. PMID: 33949931 Review.
Gene therapy is a technique that aims at the delivery of nucleic acids to cells, to obtain a therapeutic effect. ...Gene therapy finally seems to blossom as a viable therapeutic approach. The growth in the number of clinical protocols shown here is evi …
Gene therapy is a technique that aims at the delivery of nucleic acids to cells, to obtain a therapeutic effect. ...Gene …
Gene therapy for kidney disease: targeting cystinuria.
Peek JL, Wilson MH. Peek JL, et al. Curr Opin Nephrol Hypertens. 2022 Mar 1;31(2):175-179. doi: 10.1097/MNH.0000000000000768. Curr Opin Nephrol Hypertens. 2022. PMID: 34982522 Free PMC article. Review.
RECENT FINDINGS: Despite the advances of gene therapy for treating diseases of other organs, the kidney lags behind. Kidney-targeted gene delivery remains an obstacle to gene therapy of kidney disease. Nanoparticle and adeno-associated viral vector tec …
RECENT FINDINGS: Despite the advances of gene therapy for treating diseases of other organs, the kidney lags behind. Kidney-ta …
Hemophilia gene therapy-New country initiatives.
Reiss UM, Zhang L, Ohmori T. Reiss UM, et al. Haemophilia. 2021 Feb;27 Suppl 3:132-141. doi: 10.1111/hae.14080. Epub 2020 Jul 7. Haemophilia. 2021. PMID: 32638467 Review.
It is expected that the first FVIII and FIX gene therapy products will soon be approved and distributed in major markets. Global access to gene therapy is a critical goal. ...Other countries are in earlier phases of building gene therapy …
It is expected that the first FVIII and FIX gene therapy products will soon be approved and distributed in major markets. Glob …
Application of Gene Therapy in Hemophilia.
Hu YF, Fang YH, Lai YR, Feng XQ, Xu SQ. Hu YF, et al. Curr Med Sci. 2022 Oct;42(5):925-931. doi: 10.1007/s11596-022-2645-x. Epub 2022 Oct 19. Curr Med Sci. 2022. PMID: 36260269
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. ...More trials and evidence are needed to elucidate the safety and accuracy of gene therapy. This pa …
A great number of clinical trials of gene therapy in hemophilia have been carried out using viral vectors to introduce foreign …
Gene therapy for neurological disorders.
Choong CJ, Baba K, Mochizuki H. Choong CJ, et al. Expert Opin Biol Ther. 2016;16(2):143-59. doi: 10.1517/14712598.2016.1114096. Epub 2015 Dec 5. Expert Opin Biol Ther. 2016. PMID: 26642082 Review.
AREAS COVERED: This review discusses the currently available gene therapy techniques, potential problems derived from gene therapy strategies and the recent development of gene therapy to treat neurological disorders, including Alzheimer' …
AREAS COVERED: This review discusses the currently available gene therapy techniques, potential problems derived from gene …
Gene therapy for hemophilia, a clinical viewpoint.
Chou SC, Hsu YC, Lin SW. Chou SC, et al. J Formos Med Assoc. 2023 Nov;122(11):1101-1110. doi: 10.1016/j.jfma.2023.05.008. Epub 2023 May 18. J Formos Med Assoc. 2023. PMID: 37210312 Free article. Review.
The current status of gene therapy, focusing particularly on products likely to be clinically available soon, are reviewed and summarized. Currently, possible limitations of gene therapy are pre-existing neutralizing antibodies toward the vector, liver …
The current status of gene therapy, focusing particularly on products likely to be clinically available soon, are reviewed and …
Gene therapy for cardiac arrhythmias.
Bains S, Giudicessi JR, Odening KE, Ackerman MJ. Bains S, et al. Nat Rev Cardiol. 2025 May 23. doi: 10.1038/s41569-025-01168-5. Online ahead of print. Nat Rev Cardiol. 2025. PMID: 40410593 Review.
Cardiovascular diseases are the leading cause of death globally, with cardiac arrhythmias contributing substantially to this burden. Gene therapy, which directly targets the underlying disease pathobiology, offers an appealing treatment strategy for cardiac arrhythm …
Cardiovascular diseases are the leading cause of death globally, with cardiac arrhythmias contributing substantially to this burden. Gene …
Gene Therapy Approaches to Biological Pacemakers.
Farraha M, Kumar S, Chong J, Cho HC, Kizana E. Farraha M, et al. J Cardiovasc Dev Dis. 2018 Oct 19;5(4):50. doi: 10.3390/jcdd5040050. J Cardiovasc Dev Dis. 2018. PMID: 30347716 Free PMC article. Review.
With an improved understanding of the molecular mechanisms driving pacemaker development, several gene therapy targets have been identified to generate the phenotypic changes required to correct pacemaker dysfunction. This review will discuss the gene ther …
With an improved understanding of the molecular mechanisms driving pacemaker development, several gene therapy targets have be …
Antiangiogenic Eye Gene Therapy.
Corydon TJ. Corydon TJ. Hum Gene Ther. 2015 Aug;26(8):525-37. doi: 10.1089/hum.2015.064. Hum Gene Ther. 2015. PMID: 26222377 Review.
Notably, significant advancements using gene supplementation and silencing strategies have been made in the field of ocular gene therapy, thereby pinpointing ocular gene therapy as one of the compelling "actors" bringing gene therapy to t …
Notably, significant advancements using gene supplementation and silencing strategies have been made in the field of ocular gene t …
Gene Therapy for Autoimmune Disease.
Shu SA, Wang J, Tao MH, Leung PS. Shu SA, et al. Clin Rev Allergy Immunol. 2015 Oct;49(2):163-76. doi: 10.1007/s12016-014-8451-x. Clin Rev Allergy Immunol. 2015. PMID: 25277817 Review.
Advances in understanding the immunological and molecular basis of autoimmune diseases have made gene therapy a promising approach to treat the affected patients. Gene therapy for autoimmune diseases aims to regulate the levels of proinflammatory cytok …
Advances in understanding the immunological and molecular basis of autoimmune diseases have made gene therapy a promising appr …
Gene therapy for polygenic or complex diseases.
Wu T, Hu Y, Tang LV. Wu T, et al. Biomark Res. 2024 Sep 4;12(1):99. doi: 10.1186/s40364-024-00618-5. Biomark Res. 2024. PMID: 39232780 Free PMC article. Review.
Over the past two decades, significant advancements have been made in the field of gene therapy, leading to the approval of various gene therapy drugs. Gene therapy was initially employed for treating genetic diseases and cancers, particu …
Over the past two decades, significant advancements have been made in the field of gene therapy, leading to the approval of va …
Gene therapy for skin diseases.
Gorell E, Nguyen N, Lane A, Siprashvili Z. Gorell E, et al. Cold Spring Harb Perspect Med. 2014 Apr 1;4(4):a015149. doi: 10.1101/cshperspect.a015149. Cold Spring Harb Perspect Med. 2014. PMID: 24692191 Free PMC article. Review.
The skin possesses qualities that make it desirable for gene therapy, and studies have focused on gene therapy for multiple cutaneous diseases. Gene therapy uses a vector to introduce genetic material into cells to alter gene expression, …
The skin possesses qualities that make it desirable for gene therapy, and studies have focused on gene therapy f …
Gene therapy targets epilepsy.
Drew L. Drew L. Nature. 2018 Dec;564(7735):S10-S11. doi: 10.1038/d41586-018-07644-y. Nature. 2018. PMID: 30542182 No abstract available.
The ethics of gene therapy for hemophilia: a narrative review.
Baas L, van der Graaf R, van Hoorn ES, Bredenoord AL, Meijer K; SYMPHONY consortium. Baas L, et al. J Thromb Haemost. 2023 Mar;21(3):413-420. doi: 10.1016/j.jtha.2022.12.027. Epub 2023 Jan 2. J Thromb Haemost. 2023. PMID: 36696181 Free article. Review.
Gene therapy is expected to become a promising treatment, and potentially even a cure, for hemophilia. ...The theme Costs and access discusses the expected cost-effectiveness of gene therapy and its implications on accessibility worldwide. We conclude …
Gene therapy is expected to become a promising treatment, and potentially even a cure, for hemophilia. ...The theme Costs and …
[Gene therapy: Where are we? Where are we going?].
Expósito J, Natera D, Carrera L, Armijo J, Rios A, Nascimento A, Ortez C. Expósito J, et al. Medicina (B Aires). 2023 Sep;83 Suppl 4:13-17. Medicina (B Aires). 2023. PMID: 37714117 Free article. Review. Spanish.
Gene therapy has achieved significant advancements in the treatment of genetic diseases, especially in rare and monogenic diseases. ...Research on gene therapy vectors is expected to enhance the delivery capacity and safety of treatments. ...
Gene therapy has achieved significant advancements in the treatment of genetic diseases, especially in rare and monogenic dise …
Gene therapy in pancreatic cancer.
Liu SX, Xia ZS, Zhong YQ. Liu SX, et al. World J Gastroenterol. 2014 Oct 7;20(37):13343-68. doi: 10.3748/wjg.v20.i37.13343. World J Gastroenterol. 2014. PMID: 25309069 Free PMC article. Review.
Combining different targets or combination strategies with traditional chemoradiotherapy may be a more effective approach to improve the efficacy of cancer gene therapy. Cancer gene therapy is not yet applied in clinical practice, but basic and clinica …
Combining different targets or combination strategies with traditional chemoradiotherapy may be a more effective approach to improve the eff …
Gene therapy for malignant glioma.
Okura H, Smith CA, Rutka JT. Okura H, et al. Mol Cell Ther. 2014 Jul 8;2:21. doi: 10.1186/2052-8426-2-21. eCollection 2014. Mol Cell Ther. 2014. PMID: 26056588 Free PMC article. Review.
In this review, we will discuss the most commonly studied gene therapy approaches for the treatment of GBM in preclinical and clinical studies including: prodrug/suicide gene therapy; oncolytic gene therapy; cytokine mediated gene …
In this review, we will discuss the most commonly studied gene therapy approaches for the treatment of GBM in preclinical and …
[Gene therapy for primary immunodeficiency].
Onodera M. Onodera M. Rinsho Ketsueki. 2019;60(9):1358-1365. doi: 10.11406/rinketsu.60.1358. Rinsho Ketsueki. 2019. PMID: 31597864 Review. Japanese.
The former definition of gene therapy was the infusion of genes or cells transduced with genes into humans for the treatment or prevention of a disease, i.e., gene therapy adds a functional gene into the patients' genome, whereas the mutated gene remai …
The former definition of gene therapy was the infusion of genes or cells transduced with genes into humans for the treatment o …
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