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Wyszukujesz frazę ""GENE therapy"" wg kryterium: Temat


Starter badań:

Tytuł:
Expression and processing of mature human frataxin after gene therapy in mice.
Autorzy:
Rojsajjakul, Teerapat (AUTHOR)
Selvan, Nithya (AUTHOR)
De, Bishnu (AUTHOR)
Rosenberg, Jonathan B. (AUTHOR)
Kaminsky, Stephen M. (AUTHOR)
Sondhi, Dolan (AUTHOR)
Janki, Peter (AUTHOR)
Crystal, Ronald G. (AUTHOR)
Mesaros, Clementina (AUTHOR)
Khanna, Richie (AUTHOR)
Blair, Ian A. (AUTHOR)
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Źródło:
Scientific Reports. 4/10/2024, Vol. 14 Issue 1, p1-14. 14p.
Czasopismo naukowe
Tytuł:
Chitosan and Its Derivative-Based Nanoparticles in Gastrointestinal Cancers: Molecular Mechanisms of Action and Promising Anticancer Strategies.
Autorzy:
Shokati Eshkiki, Zahra
Mansouri, Fatemeh
Karamzadeh, Amir Reza
Namazi, Abolfazl
Heydari, Hafez
Akhtari, Javad
Tabaeian, Seidamir Pasha
Akbari, Abolfazl
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Źródło:
Journal of Clinical Pharmacy & Therapeutics. 4/5/2024, Vol. 2024, p1-19. 19p.
Czasopismo naukowe
Tytuł:
Identification of the Efficient Enhancer Elements in FVIII-Padua for Gene Therapy Study of Hemophilia A.
Autorzy:
Xiao, Rou (AUTHOR)
Chen, Yan (AUTHOR)
Hu, Zhiqing (AUTHOR)
Tang, Qiyu (AUTHOR)
Wang, Peiyun (AUTHOR)
Zhou, Miaojin (AUTHOR)
Wu, Lingqian (AUTHOR)
Liang, Desheng (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Apr2024, Vol. 25 Issue 7, p3635. 21p.
Czasopismo naukowe
Tytuł:
AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness.
Autorzy:
Qi, Jieyu (AUTHOR)
Tan, Fangzhi (AUTHOR)
Zhang, Liyan (AUTHOR)
Lu, Ling (AUTHOR)
Zhang, Shanzhong (AUTHOR)
Zhai, Yabo (AUTHOR)
Lu, Yicheng (AUTHOR)
Qian, Xiaoyun (AUTHOR)
Dong, WenXiu (AUTHOR)
Zhou, Yinyi (AUTHOR)
Zhang, Ziyu (AUTHOR)
Yang, Xuehan (AUTHOR)
Jiang, Lulu (AUTHOR)
Yu, Chaorong (AUTHOR)
Liu, Jiancheng (AUTHOR)
Chen, Tian (AUTHOR)
Wu, Lianqiu (AUTHOR)
Tan, Chang (AUTHOR)
Sun, Sijie (AUTHOR)
Song, Huaien (AUTHOR)
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Źródło:
Advanced Science. 3/20/2024, Vol. 11 Issue 11, p1-13. 13p.
Czasopismo naukowe
Tytuł:
Safety and Efficacy of IL-12 Plasmid DNA Transfection into Pig Skin: Supportive Data for Human Clinical Trials on Gene Therapy and Vaccination.
Autorzy:
Lampreht Tratar, Ursa (AUTHOR)
Jesenko, Tanja (AUTHOR)
Omerzel, Masa (AUTHOR)
Seliskar, Alenka (AUTHOR)
Stupan, Urban (AUTHOR)
Djokic, Mihajlo (AUTHOR)
Sredensek, Jerneja (AUTHOR)
Trotovsek, Blaz (AUTHOR)
Sersa, Gregor (AUTHOR)
Cemazar, Maja (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Mar2024, Vol. 25 Issue 6, p3151. 12p.
Czasopismo naukowe
Tytuł:
Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care.
Autorzy:
Henderson, Marian L. (AUTHOR)
Zieba, Jacob K. (AUTHOR)
Li, Xiaopeng (AUTHOR)
Campbell, Daniel B. (AUTHOR)
Williams, Michael R. (AUTHOR)
Vogt, Daniel L. (AUTHOR)
Bupp, Caleb P. (AUTHOR)
Edgerly, Yvonne M. (AUTHOR)
Rajasekaran, Surender (AUTHOR)
Hartog, Nicholas L. (AUTHOR)
Prokop, Jeremy W. (AUTHOR)
Krueger, Jena M. (AUTHOR)
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Źródło:
BioTech. Mar2024, Vol. 13 Issue 1, p1. 31p.
Czasopismo naukowe
Tytuł:
Targeted Suicide Gene Therapy with Retroviral Replicating Vectors for Experimental Canine Cancers.
Autorzy:
Sonoda-Fukuda, Emiko (AUTHOR)
Takeuchi, Yuya (AUTHOR)
Ogawa, Nao (AUTHOR)
Noguchi, Shunsuke (AUTHOR)
Takarada, Toru (AUTHOR)
Kasahara, Noriyuki (AUTHOR)
Kubo, Shuji (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Mar2024, Vol. 25 Issue 5, p2657. 11p.
Czasopismo naukowe
Tytuł:
Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes.
Autorzy:
Kolesnik, Valeria V. (AUTHOR)
Nurtdinov, Ruslan F. (AUTHOR)
Oloruntimehin, Ezekiel Sola (AUTHOR)
Karabelsky, Alexander V. (AUTHOR)
Malogolovkin, Alexander S. (AUTHOR)
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Źródło:
Clinical & Translational Medicine. Mar2024, Vol. 14 Issue 3, p1-22. 22p.
Czasopismo naukowe
Tytuł:
AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR -Associated X-Linked Retinitis Pigmentosa.
Autorzy:
Sladen, Paul E. (AUTHOR)
Naeem, Arifa (AUTHOR)
Adefila-Ideozu, Toyin (AUTHOR)
Vermeule, Tijmen (AUTHOR)
Busson, Sophie L. (AUTHOR)
Michaelides, Michel (AUTHOR)
Naylor, Stuart (AUTHOR)
Forbes, Alexandria (AUTHOR)
Lane, Amelia (AUTHOR)
Georgiadis, Anastasios (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Feb2024, Vol. 25 Issue 3, p1839. 14p.
Czasopismo naukowe
Tytuł:
Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15.
Autorzy:
Chaplin, Marty
Bresnahan, Rebecca
Fleeman, Nigel
Mahon, James
Houten, Rachel
Beale, Sophie
Boland, Angela
Dundar, Yenal
Marsden, Ashley
Munot, Pinki
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Źródło:
PharmacoEconomics - Open. Nov2023, Vol. 7 Issue 6, p863-875. 13p.
Czasopismo naukowe
Tytuł:
A position statement on the post gene-therapy rehabilitation of aromatic I-amino acid decarboxylase deficiency patients.
Autorzy:
Lee, Hui-Min (AUTHOR)
Mercimek-Andrews, Saadet (AUTHOR)
Horvath, Gabriella (AUTHOR)
Marchese, Diana (AUTHOR)
Poulin III, Richard E. (AUTHOR)
Krolick, Alexis (AUTHOR)
Tierney, Kati-Lyn (AUTHOR)
Turna, Jasmine (AUTHOR)
Wei, Judy (AUTHOR)
Hwu, Wuh-Liang (AUTHOR)
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Źródło:
Orphanet Journal of Rare Diseases. 1/18/2024, Vol. 19 Issue 1, p1-16. 16p.
Czasopismo naukowe
Tytuł:
The Road towards Gene Therapy for X-Linked Juvenile Retinoschisis: A Systematic Review of Preclinical Gene Therapy in Cell-Based and Rodent Models of XLRS.
Autorzy:
van der Veen, Isa (AUTHOR)
Heredero Berzal, Andrea (AUTHOR)
Koster, Céline (AUTHOR)
ten Asbroek, Anneloor L. M. A. (AUTHOR)
Bergen, Arthur A. (AUTHOR)
Boon, Camiel J. F. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Jan2024, Vol. 25 Issue 2, p1267. 37p.
Czasopismo naukowe
Tytuł:
The Clinical Findings, Pathogenic Variants, and Gene Therapy Qualifications Found in a Leber Congenital Amaurosis Phenotypic Spectrum Patient Cohort.
Autorzy:
Sather III, Richard (AUTHOR)
Ihinger, Jacie (AUTHOR)
Simmons, Michael (AUTHOR)
Lobo, Glenn P. (AUTHOR)
Montezuma, Sandra R. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Jan2024, Vol. 25 Issue 2, p1253. 16p.
Czasopismo naukowe
Tytuł:
Recent advances in various adeno-associated viruses (AAVs) as gene therapy agents in hepatocellular carcinoma.
Autorzy:
Hadi, Meead (AUTHOR)
Qutaiba B. Allela, Omer (AUTHOR)
Jabari, Mansoureh (AUTHOR)
Jasoor, Asna Mahyazadeh (AUTHOR)
Naderloo, Omid (AUTHOR)
Yasamineh, Saman (AUTHOR)
Gholizadeh, Omid (AUTHOR)
Kalantari, Leila (AUTHOR)
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Źródło:
Virology Journal. 1/12/2024, Vol. 21 Issue 1, p1-22. 22p.
Czasopismo naukowe
Tytuł:
Serum-Resistant Ternary DNA Polyplexes for Suicide Gene Therapy of Uterine Leiomyoma.
Autorzy:
Egorova, Anna (AUTHOR)
Shtykalova, Sofia (AUTHOR)
Maretina, Marianna (AUTHOR)
Freund, Svetlana (AUTHOR)
Selutin, Alexander (AUTHOR)
Shved, Natalia (AUTHOR)
Selkov, Sergei (AUTHOR)
Kiselev, Anton (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Jan2024, Vol. 25 Issue 1, p34. 29p.
Czasopismo naukowe
Tytuł:
Gene Therapy for Inherited Retinal Diseases: From Laboratory Bench to Patient Bedside and Beyond.
Autorzy:
Brar, Anand Singh (AUTHOR)
Parameswarappa, Deepika C. (AUTHOR)
Takkar, Brijesh (AUTHOR)
Narayanan, Raja (AUTHOR)
Jalali, Subhadra (AUTHOR)
Mandal, Sohini (AUTHOR)
Fujinami, Kaoru (AUTHOR)
Padhy, Srikanta Kumar (AUTHOR)
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Źródło:
Ophthalmology & Therapy. Jan2024, Vol. 13 Issue 1, p21-50. 30p.
Czasopismo naukowe
Tytuł:
Dual peptides-modified cationic liposomes for enhanced Lung cancer gene therapy by a gap junction regulating strategy.
Autorzy:
Zhao, Ziyu (AUTHOR)
Wang, Wenhao (AUTHOR)
Wang, Guanlin (AUTHOR)
Huang, Zhengwei (AUTHOR)
Zhou, Liping (AUTHOR)
Lin, Li (AUTHOR)
Ou, Yueling (AUTHOR)
Huang, Wanzhen (AUTHOR)
Zhang, Xuejuan (AUTHOR)
Wu, Chuanbin (AUTHOR)
Tao, Liang (AUTHOR)
Wang, Qin (AUTHOR)
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Źródło:
Journal of Nanobiotechnology. 12/9/2023, Vol. 21 Issue 1, p1-17. 17p.
Czasopismo naukowe
Tytuł:
Mitochondrially Targeted Gene Therapy Rescues Visual Loss in a Mouse Model of Leber's Hereditary Optic Neuropathy.
Autorzy:
Chou, Tsung-Han (AUTHOR)
Hao, Zixuan (AUTHOR)
Alba, Diego (AUTHOR)
Lazo, Angelina (AUTHOR)
Gallo Afflitto, Gabriele (AUTHOR)
Eastwood, Jeremy D. (AUTHOR)
Porciatti, Vittorio (AUTHOR)
Guy, John (AUTHOR)
Yu, Hong (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Dec2023, Vol. 24 Issue 23, p17068. 14p.
Czasopismo naukowe
Tytuł:
Adenoviral Gene Therapy Vectors in Clinical Use—Basic Aspects with a Special Reference to Replication-Competent Adenovirus Formation and Its Impact on Clinical Safety.
Autorzy:
Leikas, Aleksi J. (AUTHOR)
Ylä-Herttuala, Seppo (AUTHOR)
Hartikainen, Juha E. K. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Nov2023, Vol. 24 Issue 22, p16519. 20p.
Czasopismo naukowe
Tytuł:
AAV-SPL 2.0, a Modified Adeno-Associated Virus Gene Therapy Agent for the Treatment of Sphingosine Phosphate Lyase Insufficiency Syndrome.
Autorzy:
Khan, Ranjha (AUTHOR)
Oskouian, Babak (AUTHOR)
Lee, Joanna Y. (AUTHOR)
Hodgin, Jeffrey B. (AUTHOR)
Yang, Yingbao (AUTHOR)
Tassew, Gizachew (AUTHOR)
Saba, Julie D. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Nov2023, Vol. 24 Issue 21, p15560. 16p.
Czasopismo naukowe
Tytuł:
Gene therapy for alopecia in type II rickets model rats using vitamin D receptor-expressing adenovirus vector.
Autorzy:
Kise, Satoko (AUTHOR)
Iijima, Ayano (AUTHOR)
Nagao, Chika (AUTHOR)
Okada, Tadashi (AUTHOR)
Nishikawa, Miyu (AUTHOR)
Ikushiro, Shinichi (AUTHOR)
Nakanishi, Tomoko (AUTHOR)
Sato, Shigeto (AUTHOR)
Yasuda, Kaori (AUTHOR)
Sakaki, Toshiyuki (AUTHOR)
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Źródło:
Scientific Reports. 10/28/2023, Vol. 13 Issue 1, p1-10. 10p.
Czasopismo naukowe
Tytuł:
CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene Therapy.
Autorzy:
Geoffroy, Marine (AUTHOR)
Pili, Louna (AUTHOR)
Buffa, Valentina (AUTHOR)
Caroff, Maëlle (AUTHOR)
Bigot, Anne (AUTHOR)
Gicquel, Evelyne (AUTHOR)
Rouby, Grégory (AUTHOR)
Richard, Isabelle (AUTHOR)
Fragnoud, Romain (AUTHOR)
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Źródło:
Cells (2073-4409). Oct2023, Vol. 12 Issue 20, p2444. 16p.
Czasopismo naukowe
Tytuł:
Retroviral Replicating Vectors Mediated Prodrug Activator Gene Therapy in a Gastric Cancer Model.
Autorzy:
Fujino, Hiroaki (AUTHOR)
Sonoda-Fukuda, Emiko (AUTHOR)
Isoda, Lisa (AUTHOR)
Kawabe, Ayane (AUTHOR)
Takarada, Toru (AUTHOR)
Kasahara, Noriyuki (AUTHOR)
Kubo, Shuji (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Oct2023, Vol. 24 Issue 19, p14823. 11p.
Czasopismo naukowe
Tytuł:
Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis.
Autorzy:
Vyas, Meera (AUTHOR)
Deschenes, Natalie M. (AUTHOR)
Osmon, Karlaina J. L. (AUTHOR)
Chen, Zhilin (AUTHOR)
Ahmad, Imtiaz (AUTHOR)
Kot, Shalini (AUTHOR)
Thompson, Patrick (AUTHOR)
Richmond, Chris (AUTHOR)
Gray, Steven J. (AUTHOR)
Walia, Jagdeep S. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Oct2023, Vol. 24 Issue 19, p14611. 15p.
Czasopismo naukowe
Tytuł:
EpiPro, a Novel, Synthetic, Activity-Regulated Promoter That Targets Hyperactive Neurons in Epilepsy for Gene Therapy Applications.
Autorzy:
Burke, Cassidy T. (AUTHOR)
Vitko, Iuliia (AUTHOR)
Straub, Justyna (AUTHOR)
Nylund, Elsa O. (AUTHOR)
Gawda, Agnieszka (AUTHOR)
Blair, Kathryn (AUTHOR)
Sullivan, Kyle A. (AUTHOR)
Ergun, Lara (AUTHOR)
Ottolini, Matteo (AUTHOR)
Patel, Manoj K. (AUTHOR)
Perez-Reyes, Edward (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Oct2023, Vol. 24 Issue 19, p14467. 21p.
Czasopismo naukowe
Tytuł:
Gene Therapy in Hereditary Retinal Dystrophies: The Usefulness of Diagnostic Tools in Candidate Patient Selections.
Autorzy:
Malvasi, Mariaelena (AUTHOR)
Casillo, Lorenzo (AUTHOR)
Avogaro, Filippo (AUTHOR)
Abbouda, Alessandro (AUTHOR)
Vingolo, Enzo Maria (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Sep2023, Vol. 24 Issue 18, p13756. 32p.
Czasopismo naukowe
Tytuł:
A phase I clinical trial of oncolytic adenovirus mediated suicide and interleukin-12 gene therapy in patients with recurrent localized prostate adenocarcinoma.
Autorzy:
Nyati, Shyam (AUTHOR)
Stricker, Hans (AUTHOR)
Barton, Kenneth N. (AUTHOR)
Li, Pin (AUTHOR)
Elshaikh, Mohamed (AUTHOR)
Ali, Haythem (AUTHOR)
Brown, Stephen L. (AUTHOR)
Hwang, Clara (AUTHOR)
Peabody, James (AUTHOR)
Freytag, Svend O. (AUTHOR)
Movsas, Benjamin (AUTHOR)
Siddiqui, Farzan (AUTHOR)
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Źródło:
PLoS ONE. 9/15/2023, Vol. 18 Issue 9, p1-22. 22p.
Czasopismo naukowe
Tytuł:
Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape.
Autorzy:
Ponomarev, Aleksei S. (AUTHOR)
Chulpanova, Daria S. (AUTHOR)
Yanygina, Lina M. (AUTHOR)
Solovyeva, Valeriya V. (AUTHOR)
Rizvanov, Albert A. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Sep2023, Vol. 24 Issue 18, p13743. 21p.
Czasopismo naukowe
Tytuł:
Systematic gene therapy derived from an investigative study of AAV2/8 vector gene therapy for Fabry disease.
Autorzy:
Deng, Mulan (AUTHOR)
Zhou, Hongyu (AUTHOR)
He, Shaomei (AUTHOR)
Qiu, Haoheng (AUTHOR)
Wang, Yanping (AUTHOR)
Zhao, April Yuanyi (AUTHOR)
Mu, Yunping (AUTHOR)
Li, Fanghong (AUTHOR)
Zhao, Allan Zijian (AUTHOR)
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Źródło:
Orphanet Journal of Rare Diseases. 9/5/2023, Vol. 18 Issue 1, p1-13. 13p.
Czasopismo naukowe
Tytuł:
Liposomal delivery of gene therapy for ovarian cancer: a systematic review.
Autorzy:
Son, Jin Sung (AUTHOR)
Chow, Ryan (AUTHOR)
Kim, Helena (AUTHOR)
Lieu, Toney (AUTHOR)
Xiao, Maria (AUTHOR)
Kim, Sunny (AUTHOR)
Matuszewska, Kathy (AUTHOR)
Pereira, Madison (AUTHOR)
Nguyen, David Le (AUTHOR)
Petrik, Jim (AUTHOR)
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Źródło:
Reproductive Biology & Endocrinology. 8/23/2023, Vol. 21 Issue 1, p1-11. 11p.
Czasopismo naukowe
Tytuł:
Bioorthogonal Engineered Virus-Like Nanoparticles for Efficient Gene Therapy.
Autorzy:
Bao, Chun-Jie (AUTHOR)
Duan, Jia-Lun (AUTHOR)
Xie, Ying (AUTHOR)
Feng, Xin-Ping (AUTHOR)
Cui, Wei (AUTHOR)
Chen, Song-Yue (AUTHOR)
Li, Pei-Shan (AUTHOR)
Liu, Yi-Xuan (AUTHOR)
Wang, Jin-Ling (AUTHOR)
Wang, Gui-Ling (AUTHOR)
Lu, Wan-Liang (AUTHOR)
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Źródło:
Nano-Micro Letters. 8/12/2023, Vol. 15 Issue 1, p1-17. 17p.
Czasopismo naukowe
Tytuł:
Mapping brain networks in MPS I mice and their restoration following gene therapy.
Autorzy:
Zhu, Wei (AUTHOR)
Ou, Li (AUTHOR)
Zhang, Lin (AUTHOR)
Clark, Isaac H. (AUTHOR)
Zhang, Ying (AUTHOR)
Zhu, Xiao-Hong (AUTHOR)
Whitley, Chester B. (AUTHOR)
Hackett, Perry B. (AUTHOR)
Low, Walter C. (AUTHOR)
Chen, Wei (AUTHOR)
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Źródło:
Scientific Reports. 8/5/2023, Vol. 13 Issue 1, p1-11. 11p.
Czasopismo naukowe
Tytuł:
TGF β Inhibitor A83-01 Enhances Murine HSPC Expansion for Gene Therapy.
Autorzy:
Fleischauer, Jenni (AUTHOR)
Bastone, Antonella Lucia (AUTHOR)
Selich, Anton (AUTHOR)
John-Neek, Philipp (AUTHOR)
Weisskoeppel, Luisa (AUTHOR)
Schaudien, Dirk (AUTHOR)
Schambach, Axel (AUTHOR)
Rothe, Michael (AUTHOR)
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Źródło:
Cells (2073-4409). Aug2023, Vol. 12 Issue 15, p1978. 25p.
Czasopismo naukowe
Tytuł:
Immunostimulatory gene therapy targeting CD40, 4-1BB and IL-2R activates DCs and stimulates antigen-specific T-cell and NK-cell responses in melanoma models.
Autorzy:
Wenthe, Jessica (AUTHOR)
Eriksson, Emma (AUTHOR)
Hellström, Ann-Charlotte (AUTHOR)
Moreno, Rafael (AUTHOR)
Ullenhag, Gustav (AUTHOR)
Alemany, Ramon (AUTHOR)
Lövgren, Tanja (AUTHOR)
Loskog, Angelica (AUTHOR)
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Źródło:
Journal of Translational Medicine. 7/27/2023, Vol. 21 Issue 1, p1-17. 17p.
Czasopismo naukowe
Tytuł:
Drug-Inducible Gene Therapy Effectively Reduces Spontaneous Seizures in Kindled Rats but Creates Off-Target Side Effects in Inhibitory Neurons.
Autorzy:
Sullivan, Kyle A. (AUTHOR)
Vitko, Iuliia (AUTHOR)
Blair, Kathryn (AUTHOR)
Gaykema, Ronald P. (AUTHOR)
Failor, Madison J. (AUTHOR)
San Pietro, Jennifer M. (AUTHOR)
Dey, Deblina (AUTHOR)
Williamson, John M. (AUTHOR)
Stornetta, Ruth L. (AUTHOR)
Kapur, Jaideep (AUTHOR)
Perez-Reyes, Edward (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Jul2023, Vol. 24 Issue 14, p11347. 17p.
Czasopismo naukowe
Tytuł:
Adenovirus-Based Gene Therapy for Bone Regeneration: A Comparative Analysis of In Vivo and Ex Vivo BMP2 Gene Delivery.
Autorzy:
Bukharova, Tatiana Borisovna (AUTHOR)
Nedorubova, Irina Alekseevna (AUTHOR)
Mokrousova, Viktoria Olegovna (AUTHOR)
Meglei, Anastasiia Yurevna (AUTHOR)
Basina, Viktoriia Pavlovna (AUTHOR)
Nedorubov, Andrey Anatolevich (AUTHOR)
Vasilyev, Andrey Vyacheslavovich (AUTHOR)
Grigoriev, Timofei Evgenevich (AUTHOR)
Zagoskin, Yuriy Dmitrievich (AUTHOR)
Chvalun, Sergei Nicolaevich (AUTHOR)
Kutsev, Sergey Ivanovich (AUTHOR)
Goldshtein, Dmitry Vadimovich (AUTHOR)
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Źródło:
Cells (2073-4409). Jul2023, Vol. 12 Issue 13, p1762. 18p.
Czasopismo naukowe
Tytuł:
Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping.
Autorzy:
Zhu, Alex (AUTHOR)
Chiba, Shuntaro (AUTHOR)
Shimizu, Yuki (AUTHOR)
Kunitake, Katsuhiko (AUTHOR)
Okuno, Yasushi (AUTHOR)
Aoki, Yoshitsugu (AUTHOR)
Yokota, Toshifumi (AUTHOR)
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Źródło:
Pharmaceutics. Jul2023, Vol. 15 Issue 7, p1808. 13p.
Czasopismo naukowe
Tytuł:
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease.
Autorzy:
Hughes, Michael Paul (AUTHOR)
Nelvagal, Hemanth Ramesh (AUTHOR)
Coombe-Tennant, Oliver (AUTHOR)
Smith, Dave (AUTHOR)
Smith, Claire (AUTHOR)
Massaro, Giulia (AUTHOR)
Poupon-Bejuit, Laura (AUTHOR)
Platt, Frances Mary (AUTHOR)
Rahim, Ahad Abdul (AUTHOR)
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Źródło:
Cells (2073-4409). Jun2023, Vol. 12 Issue 12, p1619. 21p.
Czasopismo naukowe
Tytuł:
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Autorzy:
Bugiani, Marianna (AUTHOR)
Abbink, Truus E. M. (AUTHOR)
Edridge, Arthur W. D. (AUTHOR)
van der Hoek, Lia (AUTHOR)
Hillen, Anne E. J. (AUTHOR)
van Til, Niek P. (AUTHOR)
Hu‐A‐Ng, Gino V. (AUTHOR)
Breur, Marjolein (AUTHOR)
Aiach, Karen (AUTHOR)
Drevot, Philippe (AUTHOR)
Hocquemiller, Michaël (AUTHOR)
Laufer, Ralph (AUTHOR)
Wijburg, Frits A. (AUTHOR)
van der Knaap, Marjo S. (AUTHOR)
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Źródło:
Annals of Clinical & Translational Neurology. Jun2023, Vol. 10 Issue 6, p904-917. 14p.
Czasopismo naukowe
Tytuł:
Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs.
Autorzy:
Mullagulova, Aysilu (AUTHOR)
Shaimardanova, Alisa (AUTHOR)
Solovyeva, Valeriya (AUTHOR)
Mukhamedshina, Yana (AUTHOR)
Chulpanova, Daria (AUTHOR)
Kostennikov, Alexander (AUTHOR)
Issa, Shaza (AUTHOR)
Rizvanov, Albert (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Jun2023, Vol. 24 Issue 11, p9204. 18p.
Czasopismo naukowe
Tytuł:
How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy?
Autorzy:
Drummond, Michael (AUTHOR)
Ciani, Oriana (AUTHOR)
Fornaro, Giulia (AUTHOR)
Jommi, Claudio (AUTHOR)
Dietrich, Eva Susanne (AUTHOR)
Espin, Jaime (AUTHOR)
Mossman, Jean (AUTHOR)
de Pouvourville, Gerard (AUTHOR)
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Źródło:
BMC Health Services Research. 5/25/2023, Vol. 23 Issue 1, p1-18. 18p. 7 Charts, 1 Graph.
Czasopismo naukowe
Tytuł:
E-Selectin/AAV Gene Therapy Promotes Myogenesis and Skeletal Muscle Recovery in a Mouse Hindlimb Ischemia Model.
Autorzy:
Ribieras, Antoine J. (AUTHOR)
Ortiz, Yulexi Y. (AUTHOR)
Li, Yan (AUTHOR)
Le, Nga T. (AUTHOR)
Huerta, Carlos T. (AUTHOR)
Voza, Francesca A. (AUTHOR)
Shao, Hongwei (AUTHOR)
Vazquez-Padron, Roberto I. (AUTHOR)
Liu, Zhao-Jun (AUTHOR)
Velazquez, Omaida C. (AUTHOR)
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Źródło:
Cardiovascular Therapeutics. 5/19/2023, p1-10. 10p.
Czasopismo naukowe
Tytuł:
CRISPR-Mediated In Situ Introduction or Integration of F9-Padua in Human iPSCs for Gene Therapy of Hemophilia B.
Autorzy:
Tang, Qiyu (AUTHOR)
Hu, Zhiqing (AUTHOR)
Zhao, Junya (AUTHOR)
Zhou, Tao (AUTHOR)
Tang, Shuqing (AUTHOR)
Wang, Peiyun (AUTHOR)
Xiao, Rou (AUTHOR)
Chen, Yan (AUTHOR)
Wu, Lingqian (AUTHOR)
Zhou, Miaojin (AUTHOR)
Liang, Desheng (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. May2023, Vol. 24 Issue 10, p9013. 21p.
Czasopismo naukowe
Tytuł:
Gene Therapy Based on Mesenchymal Stem Cells Derived from Adipose Tissue for the Treatment of Obesity and Its Metabolic Complications.
Autorzy:
Lopez-Yus, Marta (AUTHOR)
García-Sobreviela, Maria Pilar (AUTHOR)
del Moral-Bergos, Raquel (AUTHOR)
Arbones-Mainar, Jose M. (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Apr2023, Vol. 24 Issue 8, p7468. 22p.
Czasopismo naukowe
Tytuł:
Effects of Human LAV-BPIFB4 Gene Therapy on the Epigenetic Clock and Health of Aged Mice.
Autorzy:
Giuliani, Maria Elisa (AUTHOR)
Barbi, Veronica (AUTHOR)
Bigossi, Giorgia (AUTHOR)
Marcozzi, Serena (AUTHOR)
Giacconi, Robertina (AUTHOR)
Cardelli, Maurizio (AUTHOR)
Piacenza, Francesco (AUTHOR)
Orlando, Fiorenza (AUTHOR)
Ciaglia, Elena (AUTHOR)
Cattaneo, Monica (AUTHOR)
Mongelli, Alessia (AUTHOR)
Gaetano, Carlo (AUTHOR)
Provinciali, Mauro (AUTHOR)
Puca, Annibale Alessandro (AUTHOR)
Malavolta, Marco (AUTHOR)
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Źródło:
International Journal of Molecular Sciences. Apr2023, Vol. 24 Issue 7, p6464. 11p.
Czasopismo naukowe

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