Works about GENE therapy

Results: 5000

P.116 Improving the NeuroD1-AAV-based gene therapy intracerebral injection protocol for optimal neuronal recovery.

Published in:

2025

By:

Taheri, G;
Masotti, BA;
Garcia, GR;
Wan, R;
Wilson, M;
Cook, DJ

Publication type:

Abstract

P.115 Regenerative white matter effect of neurod1-based gene therapy in non-human primate stroke model.

Published in:

2025

By:

Ramirez-Garcia, G;
Masotti, BA;
Taheri, G;
Wan, R;
Wilson, M;
Cook, DJ

Publication type:

Abstract

P.076 3-year Clinical Lessons Learned from the Alberta Spinal Muscular Atrophy Newborn Screening (SMA-NBS).

Published in:

2025

By:

Mah, JK;
Price, TR;
Crone, M;
Kolski, H;
Niri, F

Publication type:

Abstract

High-yield bioproduction of virus-free virus-like P4-EKORhE multi-lysin transducing particles as an antimicrobial gene therapeutic.

Published in:

Frontiers in Cellular & Infection Microbiology, 2025, p. 1, doi. 10.3389/fcimb.2025.1561443

By:

Ramirez-Garcia, Robert;
Sagona, Antonia P.;
Barr, Jeremy J.;
Jaramillo, Alfonso

Publication type:

Article

In vitro HIV DNA integration in STAT3 drives T cell persistence—A model of HIV-associated T cell lymphoma.

Published in:

PLoS Pathogens, 2025, v. 21, n. 7, p. 1, doi. 10.1371/journal.ppat.1013087

By:

Rist, Michael;
Kaku, Machika;
Coffin, John M.

Publication type:

Article

Correction to: Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa.

Published in:

2025

By:

Marinkovich, M. Peter;
Paller, Amy S.;
Guide, Shireen V.;
Gonzalez, Mercedes E.;
Lucky, Anne W.;
Bağcı, Işın Sinem;
Agostini, Brittani;
Fitzgerald, Kolleen;
Chen, Shijie;
Chen, Hubert;
Conner, Meghan M.;
Krishnan, Suma M.

Publication type:

Correction Notice

Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa.

Published in:

American Journal of Clinical Dermatology, 2025, v. 26, n. 4, p. 623, doi. 10.1007/s40257-025-00942-y

By:

Marinkovich, M. Peter;
Paller, Amy S.;
Guide, Shireen V.;
Gonzalez, Mercedes E.;
Lucky, Anne W.;
Bağcı, Işın Sinem;
Agostini, Brittani;
Fitzgerald, Kolleen;
Chen, Shijie;
Chen, Hubert;
Conner, Meghan M.;
Krishnan, Suma M.

Publication type:

Article

GalNAc End‐Capped Poly(β‐amino ester)s with Screened Side Chains for Efficient Gene Delivery.

Published in:

Small Methods, 2025, v. 9, n. 6, p. 1, doi. 10.1002/smtd.202401286

By:

Liu, Xinlong;
Li, Yimeng;
Liu, Xinhua;
Huang, Yangyang;
Wang, Fujun;
Li, Jiafeng;
Guo, Yuqi;
Zhu, Xinyuan;
Zhu, Lijuan;
Zhang, Chuan

Publication type:

Article

Current State of Intravesical Therapies for Nonmuscle-Invasive Bladder Cancer.

Published in:

AUANews, 2025, v. 30, n. 7, p. 13

By:

Filon, Mikolaj;
Schmidt, Bogdana

Publication type:

Article

Preclinical evaluation of the efficacy and safety of adeno-associated virus 8-tissue-nonspecific alkaline phosphatase-D10 in Alpl-/- and AlplPrx1/Prx1 mouse models for the treatment of early and late-onset hypophosphatasia

Published in:

Journal of Bone & Mineral Research, 2025, v. 40, n. 4, p. 463, doi. 10.1093/jbmr/zjaf005

By:

de Oliveira, Flavia Amadeu;
Tokuhara, Cintia Kazuko;
Mohamed, Fatma F;
Narisawa, Sonoko;
Lira dos Santos, Elis J;
Andras, Natalie L;
Shadid, Mohammad;
Miyake, Koichi;
Foster, Brian L;
Millán, José Luis

Publication type:

Article

Unraveling novel variants in the NF1 gene and investigating potential therapeutic strategies.

Published in:

Scientific Reports, 2025, v. 15, n. 1, p. 1, doi. 10.1038/s41598-025-07318-6

By:

Huang, Jianmei;
Yang, Ke;
Wang, Yaoping;
Ma, Xinrui;
Yang, Wenke;
Huo, Xiaodong;
Bai, Jie;
Zhu, Hongjie;
Wang, Jinming;
Lv, Yibing;
Liao, Shixiu

Publication type:

Article

Gene expression and hormonal signaling in osteoporosis: from molecular mechanisms to clinical breakthroughs.

Published in:

Journal of Biomaterials Science -- Polymer Edition, 2025, v. 36, n. 10, p. 1466, doi. 10.1080/09205063.2024.2445376

By:

Singh, Gurinderdeep;
Darwin, Ronald;
Panda, Krishna Chandra;
Afzal, Shaikh Amir;
Katiyar, Shashwat;
Dhakar, Ram C.;
Mani, Sangeetha

Publication type:

Article

Mice with a diverse human T cell receptor repertoire selected on multiple HLA class I molecules.

Published in:

Nature Communications, 2025, v. 16, n. 1, p. 1, doi. 10.1038/s41467-025-61306-y

By:

Dhamodaran, Arunraj;
Chen, Xiaojing;
Fellmer, Niklas;
Agrawal, Deepti;
Danner, Eric;
Kühn, Ralf;
Blankenstein, Thomas

Publication type:

Article

Viral vectors in neurodegenerative diseases: immune responses and therapeutic applications.

Published in:

Frontiers in Neurology, 2025, p. 1, doi. 10.3389/fneur.2025.1603125

By:

Wang, Yifei;
Mu, Siyu;
Liu, Fangxi

Publication type:

Article

Exploring new avenues in non--muscle-invasive bladder cancer treatment.

Published in:: 2025
Publication type:: Conference Paper/Materials

NEW NMIBC TREATMENTS FACE SLOW UPTAKE: Despite new treatment options, a survey reveals that many urologists prefer intravesical chemotherapy.

Published in:

Urology Times, 2025, v. 53, n. 6, p. 18

By:

Saylor, Benjamin P.

Publication type:

Article

Innovations in epilepsy treatment: pharmacological strategies and machine learning applications.

Published in:

Polish Journal of Neurology & Neurosurgery / Neurologia i Neurochirurgia Polska, 2025, v. 59, n. 3, p. 199, doi. 10.5603/pjnns.103446

By:

Chmielewska, Natalia;
Szyndler, Janusz

Publication type:

Article

CNS Targeted Gene Therapies.

Published in:

International Journal of High School Research, 2025, v. 7, n. 6, p. 23, doi. 10.36838/v7i6.23

By:

Guo, Ethan J.

Publication type:

Article

A Narrative Review of Current Advances and Future Changes Regarding Bladder Cancer Treatment.

Published in:

Uro (2673-4397), 2025, v. 5, n. 2, p. 11, doi. 10.3390/uro5020011

By:

Godlewski, Dominik;
Czech, Sara;
Szpara, Jakub;
Bartusik-Aebisher, Dorota;
Aebisher, David

Publication type:

Article

Nanotheranostics Revolutionizing Gene Therapy: Emerging Applications in Gene Delivery Enhancement.

Published in:

Journal of Nanotheranostics, 2025, v. 6, n. 2, p. 10, doi. 10.3390/jnt6020010

By:

Guzmán-Sastoque, Paula;
Rodríguez, Cristian F.;
Monsalve, María Camila;
Castellanos, Stiven;
Manrique-Moreno, Andrés;
Reyes, Luis H.;
Cruz, Juan C.

Publication type:

Article

An insight into cell-penetrating peptides: perspectives on design, optimization, and targeting in drug delivery systems.

Published in:

Pharmaceutical Development & Technology, 2025, v. 30, n. 5, p. 521, doi. 10.1080/10837450.2025.2505000

By:

Khakshur, Ali Asghar;
Khodaverdi, Elham;
Kamali, Hossein;
Nokhodchi, Ali

Publication type:

Article

Newborn Screening for Gaucher Disease: The New Jersey Experience.

Published in:

International Journal of Neonatal Screening (IJNS), 2025, v. 11, n. 2, p. 34, doi. 10.3390/ijns11020034

By:

Menello, Caitlin;
Pressley, Shaney;
Steffensen, Madeline;
Schmidt, Sarah;
Pedro, Helio;
Jethva, Reena;
Valdez-Gonzalez, Karen;
Adams, Darius J.;
Gupta, Punita;
King, Lorien Tambini;
Velinov, Milen;
Anderson, Sharon;
Bizargity, Peyman;
Pletcher, Beth;
Tuite, Allysa;
Kresge, Christina;
Day-Salvatore, Debra Lynn;
Kuehl, Ryan;
Ficicioglu, Can

Publication type:

Article

Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program.

Published in:

International Journal of Neonatal Screening (IJNS), 2025, v. 11, n. 2, p. 30, doi. 10.3390/ijns11020030

By:

Malvagia, Sabrina;
Bettiol, Alessandra;
Porcaro, Margherita;
Mura, Massimo;
Funghini, Silvia;
Ombrone, Daniela;
Forni, Giulia;
Scolamiero, Emanuela;
Coppi, Filippo;
Damiano, Roberta;
Cereda, Cristina;
Simonetti, Simonetta;
Lonetti, Annalisa;
Daniotti, Marta;
Caciotti, Anna;
Morrone, Amelia;
Calbi, Valeria;
Fumagalli, Francesca;
Aiuti, Alessandro;
Procopio, Elena

Publication type:

Article

Novel Immune Response Evasion Strategy to Redose Adeno-associated Viral Vectors and Prolong Survival in Surfactant Protein-B–Deficient Mice.

Published in:

American Journal of Respiratory Cell & Molecular Biology, 2025, v. 73, n. 1, p. 120, doi. 10.1165/rcmb.2024-0247OC

By:

Kang, Martin H.;
Thomas, Sylvia P.;
Westley, Caralyn;
Blouin, Thomas;
Xu, Liqun;
Chan, Ying Kai;
Lisk, Erin;
Allen, Sarah E.;
Vadivel, Arul;
Nangle, Kennedy;
Ramamurthy, Janani;
Pei, Yanlong;
Lewis, Lunndon A.;
Chiang, Jessica J.;
Romeo, Martin J.;
Vaena, Silvia;
O'Quinn, Elizabeth C.;
Schrecker, Henry D.;
Langdon, Casey G.;
Nietert, Paul J.

Publication type:

Article

In Vitro Correction of Point Mutations in the DYSF Gene Using Prime Editing.

Published in:

International Journal of Molecular Sciences, 2025, v. 26, n. 12, p. 5647, doi. 10.3390/ijms26125647

By:

Bouchard, Camille;
Rousseau, Joël;
Lamothe, Gabriel;
Dubost, Marie;
Bastrenta, Laura;
Ramezani, Sina;
Tremblay, Jacques P.

Publication type:

Article

Hammerhead Ribozymes: Structural Insights, Catalytic Mechanisms, and Cutting-Edge Applications in Synthetic Biology.

Published in:

International Journal of Molecular Sciences, 2025, v. 26, n. 12, p. 5624, doi. 10.3390/ijms26125624

By:

Wang, Liangliang;
Liu, Yan;
Xian, Xuemin;
Zhang, Haitao

Publication type:

Article

Chitosan-Based Dressing Materials for Burn Wound Healing.

Published in:

Polymers (20734360), 2025, v. 17, n. 12, p. 1647, doi. 10.3390/polym17121647

By:

Li, Shiyu;
Pan, Wenlong;
Zhang, Ming;
Song, Kailu;
Zhou, Ziqian;
Zhao, Qilong;
Li, Guang-Zhao;
Zhu, Chongyu

Publication type:

Article

A Versatile Reporter Platform for Evaluating HDR- and NHEJ-Based Genome Editing in Airway Epithelial Cell Cultures Using an rAAV Vector.

Published in:

Viruses (1999-4915), 2025, v. 17, n. 6, p. 821, doi. 10.3390/v17060821

By:

Park, Soo Yeun;
Feng, Zehua;
Zhang, Xiujuan;
Tang, Yinghua;
Richart, Donovan;
Vorhies, Kai E.;
Qiu, Jianming;
Engelhardt, John F.;
Yan, Ziying

Publication type:

Article

Progress in Pseudotyping Lentiviral Vectors Towards Cell-Specific Gene Delivery In Vivo.

Published in:

Viruses (1999-4915), 2025, v. 17, n. 6, p. 802, doi. 10.3390/v17060802

By:

Arduini, Ariana;
Katiyar, Harshita;
Liang, Chen

Publication type:

Article

Case Series of Anesthetic Management of Gene Therapy in Children With Aromatic L‐Amino Acid Decarboxylase Deficiency.

Published in:

Pediatric Anesthesia, 2025, v. 35, n. 4, p. 316, doi. 10.1111/pan.15060

By:

Clariot, Simon;
Gras, Séverine;
Goetz, Laurent;
Boulloud, Christophe;
Bonheur, Julie;
Vayssiere, Pia;
D'Hardemare, Vincent;
Ravelli, Claudia;
Dorison, Nathalie;
Devys, Jean‐Michel

Publication type:

Article

Optimizing the anesthetic care of patients with aromatic l‐amino acid decarboxylase deficiency.

Published in:

Pediatric Anesthesia, 2025, v. 35, n. 2, p. 99, doi. 10.1111/pan.15025

By:

Kanjia, Megha K.;
Jooste, Edmund H.;
Illig, Melissa;
Neifeld Capps, Jennifer;
Eisner, Christoph;
Fan, Shou Zen;
Lenarczyk, Jerzy;
Wojdacz, Rafał

Publication type:

Article

REFUTING THE RIGHT NOT TO KNOW.

Published in:

Journal of Health Care Law & Policy, 2017, v. 19, n. 1, p. 1

By:

BERKMAN, BENJAMIN E.

Publication type:

Article

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

Inhibition of Mutation and Combating the Evolution of Antibiotic Resistance.

Published in:

PLoS Biology, 2005, v. 3, n. 5, p. 1, doi. 10.1371/journal.pbio.0030176

By:

Cirz, Ryan T;
Chin, Jodie K;
Andes, David R;
de Crécy-Lagard, Valérie;
Craig, William A;
Romesberg, Floyd E

Publication type:

Article

Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences.

Published in:

PLoS Biology, 2004, v. 2, n. 8, p. 1, doi. 10.1371/journal.pbio.0020234

By:

Mitchell, Rick S;
Beitzel, Brett F;
Schroder, Astrid R. W;
Shinn, Paul;
Chen, Huaming;
Berry, Charles C;
Ecker, Joseph R;
Bushman, Frederic D

Publication type:

Article

Retrovirus Integration into the Human Genome.

Published in:

PLoS Biology, 2004, v. 2, n. 8, p. 1045, doi. 10.1371/journal.pbio.0020281

By:

RS, Mitchell;
BF, Beitzel;
SRw, Schroder;
P, Shinn;
H, Chen

Publication type:

Article

Construction and application of a novel hepatocyte-directed vector to simultaneous knockdown and overexpression of multiple genes.

Published in:

Liver International, 2014, v. 34, n. 7, p. e246, doi. 10.1111/liv.12336

By:

Geng, Jianlin;
Wei, Haiming;
Sun, Rui;
Tian, Zhigang

Publication type:

Article

Antitumoural hydroxyapatite nanoparticles-mediated hepatoma-targeted trans-arterial embolization gene therapy: in vitro and in vivo studies.

Published in:

Liver International, 2012, v. 32, n. 6, p. 998, doi. 10.1111/j.1478-3231.2012.02761.x

By:

Li, Gaopeng;
Ye, Lu;
Pan, Jingsheng;
Long, Miaoyun;
Zhao, Zizhuo;
Yang, Haiyun;
Tian, Jing;
Wen, Yanling;
Dong, Shengli;
Guan, Jian;
Luo, Baoming

Publication type:

Article

Expression and gene polymorphisms of interleukin 28B and hepatitis B virus infection in a Chinese Han population.

Published in:

Liver International, 2011, v. 31, n. 8, p. 1118, doi. 10.1111/j.1478-3231.2011.02507.x

By:

Wanyu Li;
Yanfang Jiang;
Qinglong Jin;
Xiaodong Shi;
Jinglan Jin;
Yanhang Gao;
Yu Pan;
Hong Zhang;
Jing Jiang;
Junqi Niu

Publication type:

Article

Lentivirus-mediated superoxide dismutase1 gene delivery protects against oxidative stress-induced liver injury in mice.

Published in:

Liver International, 2007, v. 27, n. 10, p. 1311, doi. 10.1111/j.1478-3231.2007.01612.x

By:

Venugopal, Senthil Kumar;
Jian Wu;
Catana, Andreea M.;
Eisenbud, Lauren;
Song-Qing He;
Yu-You Duan;
Follenzi, Antonia;
Zern, Mark A.

Publication type:

Article

IFNγ expression inhibits LHBs storage disease and ground glass hepatocyte appearance, but exacerbates inflammation and apoptosis in HBV surface protein-accumulating transgenic livers.

Published in:

Liver International, 2006, v. 26, n. 8, p. 986, doi. 10.1111/j.1478-3231.2006.01317.x

By:

Reifenberg, Kurt;
Hildt, Eberhard;
Lecher, Bernd;
Wiese, Elena;
Nusser, Petra;
Ott, Sibylle;
Yamamura, Ken-Ichi;
Rutter, Gabriel;
Löhler, Jürgen

Publication type:

Article

Portal hypertension: from pathophysiology to clinical practice.

Published in:

Liver International, 2005, v. 25, n. 6, p. 1079, doi. 10.1111/j.1478-3231.2005.01163.x

By:

Laleman, Wim;
Landeghem, Lien;
Wilmer, Alexander;
Fevery, Johan;
Nevens, Frederik

Publication type:

Article

Characteristics of murine histidinaemia and its potential for genetic manipulation.

Published in:

Liver International, 2004, v. 24, n. 4, p. 354, doi. 10.1111/j.1478-3231.2004.0929.x

By:

Mellor, N.;
Themis, M.;
Selden, C.;
Jones, M.;
Hodgson, H. J. F.

Publication type:

Article

The acceptance to germline gene therapy increased during COVID‐19 pandemic among Japanese medical students.

Published in:

2022

By:

Ijuin, Akifumi;
Hayama, Tomonari;
Sakakibara, Hideya

Publication type:

Letter to the Editor

Noninvasive approaches to peripheral vascular disease. What's new in evaluation and treatment?

Published in:

1999

By:

Powers, K B;
Vacek, J L;
Lee, S

Publication type:

journal article

Two Tuberculosis Genotyping Clusters, One Preventable Outbreak.

Published in:

Public Health Reports, 2009, v. 124, n. 4, p. 490, doi. 10.1177/003335490912400405

By:

Buff, Ann M.;
Sosa, Lynn E.;
Hoopes, Andrea J.;
Buxton-Morris, Deborah;
Condren, Thomas B.;
Hadler, James L.;
Haddad, Maryam B.;
Moonan, Patrick K.;
Lobato, Mark N.

Publication type:

Article

For A Collective Human Future Project.

Published in:

Peace Review, 2000, v. 12, n. 4, p. 579, doi. 10.1080/10402650020014672

By:

Pandikattu, Kuruvilla

Publication type:

Article

COMPUTATIONAL MODELING AND SIMULATION OF NANOMOLECULAR SWITCH FOR ALZHEIMER'S DISEASE (A GENE SILENCING TECHNIQUE).

Published in:

International Journal of Nanoscience, 2011, v. 10, n. 1/2, p. 319, doi. 10.1142/S0219581X11008010

By:

RADHAGAYATHRI, K. U.;
NAMBOORI, P. K. KRISHNAN;
MOHANDAS, V. P.;
SUBEESH, T.;
GOPAKUMAR, DEEPA;
RAMACHANDRAN, K. I.

Publication type:

Article