Works about GENE therapy

Results: 5000

Correction: Current and Emerging Therapies for Lysosomal Storage Disorders.

Published in:

2025

By:

Abelleyra Lastoria, Diego Agustín;
Keynes, Sophie;
Hughes, Derralynn

Publication type:

Correction Notice

Addressing the Gene Therapy Bottleneck in the EU: Patent vs. Regulatory Incentives.

Published in:

GRUR International: Journal of European & International IP Law, 2025, v. 74, n. 5, p. 399, doi. 10.1093/grurint/ikaf048

By:

Stierle, Martin;
Valtere, Laura

Publication type:

Article

Robust prediction of synthetic gRNA activity and cryptic DNA repair by disentangling cellular CRISPR cleavage outcomes.

Published in:

Nature Communications, 2025, v. 16, n. 1, p. 1, doi. 10.1038/s41467-025-59947-0

By:

Riesenberg, Stephan;
Kanis, Philipp;
Karlic, Rosa;
Maricic, Tomislav

Publication type:

Article

Pet godina genske terapije u hrvatskoj oftalmologiji - uspjesi, izazovi i perspektive.

Published in:

Paediatria Croatica, 2025, v. 69, p. 128, doi. 10.13112/pc.1060

By:

Bjeloš, Mirjana;
Bušić, Mladen;
Ćurić, Ana;
Bosnar, Damir;
Šarić, Borna;
Elabjer, Biljana Kuzmanović;
Predović, Jurica;
Rak, Benedict

Publication type:

Article

Gene therapy prevents onset of mitochondrial cardiomyopathy in neonatal mice with Ndufs6 deficiency.

Published in:

Cell Death Discovery, 2025, v. 11, n. 1, p. 1, doi. 10.1038/s41420-025-02524-7

By:

Zhang, Xiaoxian;
Huang, Li;
Li, Cheng;
Yang, Jinjuan;
Duan, Fuyu;
Su, Qiang;
Zhang, Yuelin;
Kou, Meng;
Zhou, Xiaoya;
Guo, Liyan;
Chen, Shaoxiang;
Niu, Yongxia;
Li, Ziyue;
Ou, Sihua;
Zhang, Min;
Cheng, Kenneth King-Yip;
Wu, Jianlong;
Xu, Xiang;
Lian, Qizhou

Publication type:

Article

A monthly roundup of key articles in other journals.

Published in:

Transfusion, 2025, v. 65, n. 5, p. 793, doi. 10.1111/trf.18250

By:

McOmish, Caitlin

Publication type:

Article

New Gene Targets for Diagnosis and Therapy of Diabetic Retinopathy.

Published in:

Eurasian Journal of Medicine, 2025, v. 57, n. 1, p. 1, doi. 10.5152/eurasianjmed.2025.24559

By:

Çinici, Emine;
Arslan, Mehmet Enes;
Yıldırım, Özge Çağlar;
Dilekmen, Nilay;
Utlu, Bahadır;
Çinici, Özkan;
Sağlam, Zehra;
Türkez, Hasan

Publication type:

Article

Mechanistic insights into PDZK1-interacting protein 1 on the malignant progression of colorectal carcinoma.

Published in:

CytoJournal, 2025, v. 22, p. 1, doi. 10.25259/Cytojournal_200_2024

By:

Yu, Kuaiyun;
Yu, Yao;
Zhang, Chang;
Hao, Leilei

Publication type:

Article

纳米粒子在乳腺癌诊断和治疗中的应用进展.

Published in:

Practical Oncology Journal, 2025, v. 39, n. 2, p. 151, doi. 10.11904/j.issn.1002-3070.2025.02.011

By:

王帅;
黎其庆;
李瑞;
许铁峰

Publication type:

Article

Exploring the expensive therapeutic potential and clinical applications of virusessevere acute respiratory syndrome (SARS)-COV-2.

Published in:

Life Research Journal, 2025, v. 8, n. 1, p. 1, doi. 10.53388/LR20250002

By:

Hejran, Abdul Bari;
Sarwari, Atiqullah;
Hassand, Mohammad Hassan;
Monib, Abdul Wahid;
Niazi, Parwiz;
Baseer, Abdul Qadeer;
Sediqi, Sayedwali;
Kakar, Uzair Mohammad

Publication type:

Article

Gene therapy: principles, challenges and use in clinical practice.

Published in:

Wiener Klinische Wochenschrift, 2025, v. 137, n. 9, p. 261, doi. 10.1007/s00508-024-02368-8

By:

Ay, Cihan;
Reinisch, Andreas

Publication type:

Article

HHS Scraps Advisory Committee on Newborn Screening.

Published in:

Neonatology Today, 2025, v. 20, n. 4, p. 178

By:

Henderson, Jennifer

Publication type:

Article

HEMOLYTIC ANEMIA: GENETIC INSIGHTS AND NEW THERAPEUTIC AVENUES.

Published in:

Obstetrics & Gynaecology Forum, 2024, v. 34, n. 3, p. 328

By:

V., Desai Jabbar;
Mane, U. T.;
Kshirsagar, N. S.

Publication type:

Article

Conditional Control of CRISPR/Cas9 Function by Chemically Modified Oligonucleotides.

Published in:

Molecules, 2025, v. 30, n. 9, p. 1956, doi. 10.3390/molecules30091956

By:

Wang, Liangliang;
Liu, Yan;
Song, Hongjun;
Zhang, Xue;
Wang, Yang

Publication type:

Article

Gene Therapy in Diabetic Retinopathy and Diabetic Macular Edema: An Update.

Published in:

Journal of Clinical Medicine, 2025, v. 14, n. 9, p. 3205, doi. 10.3390/jcm14093205

By:

Odio-Herrera, Maricruz;
Orozco-Loaiza, Gloriana;
Wu, Lihteh

Publication type:

Article

Invited Medical Presentation Abstracts.

Published in:: Haemophilia, 2025, v. 31, p. 3, doi. 10.1111/hae.70031
Publication type:: Article

mTORopathies in Epilepsy and Neurodevelopmental Disorders: The Future of Therapeutics and the Role of Gene Editing.

Published in:

Cells (2073-4409), 2025, v. 14, n. 9, p. 662, doi. 10.3390/cells14090662

By:

Boff, Marina Ottmann;
Xavier, Fernando Antônio Costa;
Diz, Fernando Mendonça;
Gonçalves, Júlia Budelon;
Ferreira, Laura Meireles;
Zambeli, Jean;
Pazzin, Douglas Bottega;
Previato, Thales Thor Ramos;
Erwig, Helena Scartassini;
Gonçalves, João Ismael Budelon;
Bruzzo, Fernanda Thays Konat;
Marinowic, Daniel;
da Costa, Jaderson Costa;
Zanirati, Gabriele

Publication type:

Article

Correction: Lactobacillus acidophilus extracellular vesiclescoated UiO-66-NH<sub>2</sub>@siRNA nanoparticles for ulcerative colitis targeted gene therapy and gut microbiota modulation.

Published in:

2025

By:

Cui, Chenyang;
Tang, Jiaze;
Chen, Jie;
Zhang, Beining;
Li, Ruonan;
Zhang, Qiang;
Qiu, Chunjing;
Chen, Rongchen;
Min, Geng;
Sun, Zhaowei;
Weng, Haibo

Publication type:

Correction Notice

Genetics in Dentistry: A Review of Advances and Implications.

Published in:

Journal of Pharmacy & Bioallied Sciences, 2025, v. 17, p. S181, doi. 10.4103/jpbs.jpbs_1758_24

By:

Singh, Jyotsana;
Salam, Sajjad;
Pasha, Zameer;
Sharma, Rohit;
Khan, Nazia;
Chunara, Farheen;
Kashwani, Ritik

Publication type:

Article

Personalized Medicine in Treating Rare Genetic Disorders: A Review.

Published in:

Journal of Pharmacy & Bioallied Sciences, 2025, v. 17, p. S59, doi. 10.4103/jpbs.jpbs_583_25

By:

Zebanaz, Ansari;
Kareem, Affan M.;
Aher, Ashwini A.;
Thitame, Sunil N.

Publication type:

Article

Large-scale discovery of potent, compact and erythroid specific enhancers for gene therapy vectors.

Published in:

Nature Communications, 2025, v. 15, n. 1, p. 1, doi. 10.1038/s41467-025-59235-x

By:

Psatha, Nikoletta;
Sova, Pavel;
Georgolopoulos, Grigorios;
Paschoudi, Kiriaki;
Iwata, Mineo;
Bloom, Jordan;
Ulyanova, Tatyana;
Wang, Hao;
Kirtsou, Alexandra;
Vasiloudis, Ninos-Ioannis;
Wilken, Matthew S.;
Stamatoyannopoulos, John A.;
Yannaki, Evangelia;
Papayanopoulou, Thalia;
Stamatoyannopoulos, George;
Vierstra, Jeff

Publication type:

Article

Gene delivery of AGAT and GAMT boosts creatine levels in creatine transporter deficiency patient fibroblasts.

Published in:

PLoS ONE, 2025, v. 20, n. 5, p. 1, doi. 10.1371/journal.pone.0319350

By:

Wells, Chloe;
Sorgenfrei, Jon;
Johnson, Sadie L.;
Albertson, Devin;
Rutter, Jared;
Baker, Steven Andrew

Publication type:

Article

The legacy of William M. Pardridge (1947–2024) on the science and fields concerned with the physiology of the blood-brain barrier and the transport of drugs to the brain.

Published in:

Fluids & Barriers of the CNS, 2025, v. 22, n. 1, p. 1, doi. 10.1186/s12987-025-00659-4

By:

Boado, Ruben J.;
Bickel, Ulrich;
Sumbria, Rachita K.

Publication type:

Article

An engineered AAV capsid targeting NK92 cells using CD56-binding peptide identified from phage display.

Published in:

Journal of Biotech Research, 2024, v. 19, p. 33

By:

Fengqi Dong;
Yunshu Hao;
Roger Wang

Publication type:

Article

Modeling Hybrid Crossover Dynamics of Immuno‐Chemotherapy and Gene Therapy: A Numerical Approach.

Published in:

Mathematical Methods in the Applied Sciences, 2025, v. 48, n. 8, p. 8925, doi. 10.1002/mma.10765

By:

Abou Hasan, Muner. M.;
AL‐Mekhlafi, Seham. M.;
Rihan, Fathala. A.;
Al‐Ali, Hannah. A.

Publication type:

Article

SCN10A-short gene therapy to restore conduction and protect against malignant cardiac arrhythmias.

Published in:

European Heart Journal, 2025, v. 46, n. 18, p. 1747, doi. 10.1093/eurheartj/ehaf053

By:

Wang, Jianan;
Verkerk, Arie O;
Wilders, Ronald;
Zhang, Yingnan;
Zhang, Kelly;
Prakosa, Adityo;
Rivaud, Mathilde R;
Marsman, E Madelief J;
Boender, Arie R;
Klerk, Mischa;
Fokkert, Lianne;
Jonge, Berend de;
Neef, Klaus;
Kirzner, Osne F;
Bezzina, Connie R;
Remme, Carol Ann;
Tan, Hanno L;
Boukens, Bastiaan J;
Devalla, Harsha D;
Trayanova, Natalia A

Publication type:

Article

Gene therapy targeting I<sub>Na</sub> to treat life-threatening arrhythmias: beyond proof-of-concept?

Published in:

European Heart Journal, 2025, v. 46, n. 18, p. 1763, doi. 10.1093/eurheartj/ehae930

By:

Lugenbiel, Patrick

Publication type:

Article

Extracellular vesicle-mediated VEGF-A mRNA delivery rescues ischaemic injury with low immunogenicity.

Published in:

European Heart Journal, 2025, v. 46, n. 17, p. 1662, doi. 10.1093/eurheartj/ehae883

By:

You, Yi;
Tian, Yu;
Guo, Rui;
Shi, Junfeng;
Kwak, Kwang Joo;
Tong, Yuhao;
Estania, Andreanne Poppy;
Hsu, Wei-Hsiang;
Liu, Yutong;
Hu, Shijun;
Cao, Jianhong;
Yang, Liqun;
Bai, Rui;
Huang, Pufeng;
Lee, Ly James;
Jiang, Wen;
Kim, Betty Y S;
Ma, Shuhong;
Liu, Xujie;
Shen, Zhenya

Publication type:

Article

Genome-wide association study and Mendelian randomization analyses reveal insights into bladder cancer etiology.

Published in:

JNCI Cancer Spectrum, 2025, v. 9, n. 2, p. 1, doi. 10.1093/jncics/pkaf014

By:

Larsson, Susanna C;
Chen, Jie;
Ruan, Xixian;
Li, Xue;
Yuan, Shuai

Publication type:

Article

Microsampling in toxicology studies – maximising the scientific, business and 3Rs advantages.

Published in:

Toxicology Research, 2025, v. 14, n. 2, p. 1, doi. 10.1093/toxres/tfaf045

By:

Prior, Helen;
Adedeji, Adeyemi O;
Allen, Romalie;
Angus, Derek;
Baker, Daniel;
Blunt, Hollie;
Coleman, David;
Dunmore, Helen-Marie;
Passini, Elisa;
Putnam, Tara;
Rosseels, Marie-Luce;
Spooner, Neil;
Stewart, Jane;
Strepka, Carol;
Stokes, Alan;
Verhaeghe, Tom;
Wilson, Amanda;
Sewell, Fiona

Publication type:

Article

Postmarketing adverse events associated with onasemnogene abeparvovec: a real-world pharmacovigilance study.

Published in:

Orphanet Journal of Rare Diseases, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13023-025-03715-2

By:

Chen, Tianyu;
Chen, Qiying;
Ye, Jingfang;
Wu, Yuzhu;
Liu, Ting;
Zhang, Yuezhen

Publication type:

Article

Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines.

Published in:

Orphanet Journal of Rare Diseases, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13023-024-03479-1

By:

Rasmussen, Charlotte Laurfelt Munch;
Burkhart, Annette;
Moos, Torben;
Thomsen, Louiza Bohn

Publication type:

Article

Otology and Neurotology in Practice: Innovation, Patient Care, and the Role of Nurses.

Published in:

ORL-Head & Neck Nursing, 2025, v. 43, n. 2, p. 7

By:

Estheimer, Kris

Publication type:

Article

The Lived Experiences of Adults With Sickle Cell Disease Deciding Curative Therapies: A Descriptive Phenomenological Study.

Published in:

Western Journal of Nursing Research, 2025, v. 47, n. 5, p. 384, doi. 10.1177/01939459251321429

By:

Jacobs-Mcfarlane, Charleen

Publication type:

Article

Pattern of pharmacological research from the last six decades.

Published in:

2019

By:

Joshi, Rupa;
Mahendiratta, Saniya;
Prakash, Ajay;
Medhi, Bikash

Publication type:

Editorial

Gene Therapy for Pyoderma Gangrenosum: Optimal Transfection Conditions and Effect of Drugs on Gene Delivery in the HaCaT Cell Line Using Cationic Liposomes.

Published in:

Skin Pharmacology & Physiology, 2016, v. 29, n. 3, p. 119, doi. 10.1159/000444859

By:

Teagle, alexandra R.;
Birchall, James C.;
Hargest, Rachel

Publication type:

Article

The Porphyrias: Clinic, Diagnostics, Novel Investigative Tools and Evolving Molecular Therapeutic Strategies.

Published in:

Skin Pharmacology & Physiology, 2010, v. 23, n. 1, p. 18, doi. 10.1159/000257260

By:

van Serooskerken, van Tuyll;

Publication type:

Article

Stem Cells and Tissue-Engineered Skin.

Published in:

Skin Pharmacology & Physiology, 2009, v. 22, n. 2, p. 55, doi. 10.1159/000178864

By:

Charruyer;
Ghadially

Publication type:

Article

The molecular pathology of hereditary breast cancer: genetic testing and therapeutic implications.

Published in:

Modern Pathology, 2005, v. 18, n. 10, p. 1305, doi. 10.1038/modpathol.3800453

By:

Honrado, Emiliano;
Benítez, Javier;
Palacios, José

Publication type:

Article

Narratives We Organize By (Book).

Published in:

2004

By:

Hernandez, Gary

Publication type:

Book Review

The Fourth Scientific Revolution.

Published in:

Society, 1996, v. 33, n. 4, p. 9, doi. 10.1007/BF02700299

By:

Huff, Toby E.

Publication type:

Article

Nonheritable Cellular Variability Accelerates the Evolutionary Processes of Cancer.

Published in:

2012

By:

Frank, Steven A.;
Rosner, Marsha Rich

Publication type:

Essay

Engineered Common Cold Virus Helps Cultured Cystic Fibrosis Tissues Clear Mucus.

Published in:

PLoS Biology, 2009, v. 7, n. 7, p. 1, doi. 10.1371/journal.pbio.1000160

By:

Meadows, Robin

Publication type:

Article

A simplified intrathymic injection technique for mice.

Published in:

Biotechnic & Histochemistry, 2012, v. 87, n. 2, p. 140, doi. 10.3109/10520295.2011.577755

By:

Liu, LL;
Du, XM;
Wang, Z;
Wu, BJ;
Jin, M;
Xin, B;
Wang, D;
Bai, ZL

Publication type:

Article

Age at menarche and menopause is not associated with two common genetic variants in the methylenetetrahydrofolate reductase ( MTHFR) gene.

Published in:

European Journal of Contraception & Reproductive Health Care, 2011, v. 16, n. 4, p. 241, doi. 10.3109/13625187.2011.575481

By:

Cerne, Jasmina-Ziva;
Pohar-Perme, Maja;
Cerkovnik, Petra;
Gersak, Ksenija;
Novakovic, Srdjan

Publication type:

Article

Meeting Reporter.

Published in:

Patient Care (Print), 2001, v. 35, n. 11, p. 11

By:

Pinkowish, Mary Desmond;
Saunders, Carol S.

Publication type:

Article

Macular degeneration is a family affair.

Published in:: Patient Care (Print), 1998, v. 32, n. 18, p. 158
Publication type:: Article

Ethical concerns and the future of gene therapy.

Published in:: Patient Care (Print), 1998, v. 32, n. 18, p. 88
Publication type:: Article

Clinical applications of molecular medicine.

Published in:: Patient Care (Print), 1998, v. 32, n. 18, p. 86
Publication type:: Article

Gene therapy: Sci-fi no longer.

Published in:

Patient Care (Print), 1993, v. 27, n. 11, p. 24

By:

Blaese, R. Michael;
Miller, A. Dusty;
Plautz, Gregory E.;
Scriver, Charles R.;
Hart, Stephen

Publication type:

Article