Results 21 to 30 of about 7,750,425 (359)

AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation

Molecular Therapy: Methods & Clinical Development, 2021
Adeno-associated virus (AAV) vectors are considered efficient vectors for gene transfer, as illustrated by recent successful clinical trials targeting retinal or neurodegenerative disorders.
Gwladys Gernoux, Mickaël Guilbaud, Marie Devaux, Malo Journou, Virginie Pichard, Nicolas Jaulin, Adrien Léger, Johanne Le Duff, Jack-Yves Deschamps, Caroline Le Guiner, Philippe Moullier, Yan Cherel, Oumeya Adjali   +12 more
doaj   +1 more source

Cellular and genetic therapies for haemophilia [PDF]

Haemophilia, 2006
Summary.  Haemophilia continues to be a prime target for a variety of gene and cell‐based therapies. Pre‐clinical successes in both mouse and dog models of the disease have been documented with a variety of approaches over the past decade, and there have now been six small clinical trials of gene transfer in haemophilia.
Lillicrap, David, VandenDriessche, T., High, K.   +2 more
openaire   +3 more sources

Human Neural Stem Cells: Translational Research for Neonatal Hypoxic-Ischemic Brain Injury [PDF]

Neonatal Medicine, 2019
Neonatal hypoxic-ischemic (HI) brain injury is a major cause of neonatal mortality and long-term neurodevelopmental disabilities. Although promising neuroprotective interventions have been studied, the current management of HI brain injury has been ...
Jeong Eun Shin, Jungho Han, Joo Hee Lim, Ho Seon Eun, Kook In Park   +4 more
doaj   +1 more source

In Vitro Synergistic Enhancement of Newcastle Disease Virus to 5-Fluorouracil Cytotoxicity against Tumor Cells

Biomedicines, 2016
Background: Chemotherapy is one of the antitumor therapies used worldwide in spite of its serious side effects and unsatisfactory results. Many attempts have been made to increase its activity and reduce its toxicity.
Ahmed M. Al-Shammari, Marwa I. Salman, Yahya D. Saihood, Nahi Y. Yaseen, Khansaa Raed, Hiba Kareem Shaker, Aesar Ahmed, Aseel Khalid, Ahlam Duiach   +8 more
doaj   +1 more source

DNA Edition: ad portas of a revolution in genetic manipulation

Iatreia, 2020
In biological sciences, genetic therapy constitutes a “trend topic” since its beginning. Development of new technologies in bioengineering as zinc-finger nucleases (ZFN), Transcription activator-like effector nu-cleases (TALEN) and ...
Madariaga Perpiñán, Ithzayana , Duque Restrepo, Juan Camilo, Ayala Ramírez, Paola, García Robles, Reggie   +3 more
doaj   +1 more source

Refining the impact of genetic evidence on clinical success

medRxiv, 2023
The cost of drug discovery and development is driven primarily by failure, with just ~10% of clinical programs eventually receiving approval. We previously estimated that human genetic evidence doubles the success rate from clinical development to ...
E. Minikel, Jeffery L. Painter, Coco Chengliang Dong, Matthew R Nelson   +3 more
semanticscholar   +1 more source

Emerging Genetic Therapy for Sickle Cell Disease.

Annual Review of Medicine, 2019
The genetic basis of sickle cell disease (SCD) was elucidated >60 years ago, yet current therapy does not rely on this knowledge. Recent advances raise prospects for improved, and perhaps curative, treatment.
S. Orkin, D. E. Bauer
semanticscholar   +1 more source

Prenatal diagnosis and genetic etiology analysis of talipes equinovarus by chromosomal microarray analysis

BMC Medical Genomics, 2023
Background With the advancement of molecular technology, fetal talipes equinovarus (TE) is believed to be not only associated with chromosome aneuploidy, but also related to chromosomal microdeletion and microduplication.
Xiaorui Xie, Baojia Huang, Linjuan Su, Meiying Cai, Yuqin Chen, Xiaoqing Wu, Liangpu Xu   +6 more
doaj   +1 more source

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

New England Journal of Medicine, 2019
Genome sequencing is often pivotal in the diagnosis of rare diseases, but many of these conditions lack specific treatments. We describe how molecular diagnosis of a rare, fatal neurodegenerative condition led to the rational design, testing, and ...
Jinkuk Kim, Chu Hu, Christelle Moufawad Achkar, L. Black, J. Douville, A. Larson, Mary K. Pendergast, S. F. Goldkind, E. Lee, E. Lee, Ashley Kuniholm, Aubrie Soucy, J. Vaze, Nandkishore R. Belur, Kristina Fredriksen, Iva Stojkovska, A. Tsytsykova, M. Armant, Renata L DiDonato, Jaejoon Choi, L. Cornelissen, L. M. Pereira, E. Augustine, C. Genetti, K. Dies, Brenda Barton, Brenda Barton, L. Williams, L. Williams, Benjamin D. Goodlett, Bobbie L Riley, A. Pasternak, E. Berry, Kelly A Pflock, Stephen Chu, C. Reed, K. Tyndall, P. Agrawal, A. Beggs, P. Grant, D. Urion, R. Snyder, S. Waisbren, A. Poduri, P. J. Park, Al Patterson, A. Biffi, Joseph R. Mazzulli, O. Bodamer, O. Bodamer, C. Berde, Timothy W. Yu, Timothy W. Yu   +52 more
semanticscholar   +1 more source

Genetics and the Individualized Therapy of Vestibular Disorders [PDF]

Frontiers in Neurology, 2021
Background: Vestibular disorders (VDs) are a clinically divergent group of conditions that stem from pathology at the level of the inner ear, vestibulocochlear nerve, or central vestibular pathway. No etiology can be identified in the majority of patients with VDs.
Christine Mei, Hongsong Dong, Hongsong Dong, Eric Nisenbaum, Torin Thielhelm, Aida Nourbakhsh, Denise Yan, Molly Smeal, Yesha Lundberg, Michael E. Hoffer, Simon Angeli, Fred Telischi, Guohui Nie, Susan H. Blanton, Xuezhong Liu   +14 more
openaire   +3 more sources