AAV-based vectors for human diseases modeling in laboratory animals by Timur I. Aliev, Dmitry V. Yudkin

Subjects: Get full text

Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution by Florian Dunker-Seidler, Kathrin Breunig, Magdalena Haubner, Florian Sonntag, Markus Hörer, Rebecca C. Feiner

Subjects: “…nanopore sequencing, gene therapy…”
Get full text

Genetic advancements in breast cancer treatment: a review by Marzieh Shokoohi, Sadaf Sedaghatshoar, Homaira Arian, Milad Mokarami, Fatemeh Habibi, Fatemeh Bamarinejad

Subjects: Get full text

Gene Therapy: A step toward advanced orthodontics – A narrative review by Monika Shukla, Shalu Jain, C. Munish Reddy, Pradeep Raghav, Kumar Amit, Rinchin Yangzom, Shivika Garg, Aastha Kamrani

Subjects: Get full text

CRISPR-Cas9 in Cardiovascular Medicine: Unlocking New Potential for Treatment by Klaudia Bonowicz, Dominika Jerka, Klaudia Piekarska, Janet Olagbaju, Laura Stapleton, Munirat Shobowale, Andrzej Bartosiński, Magdalena Łapot, Yidong Bai, Maciej Gagat

Subjects: Get full text

Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy by Craig M. McDonald, Jacob S. Elkins, Sai Dharmarajan, Katherine Gooch, Teofil Ciobanu, Claire J. Lansdall, Alexander P. Murphy, Fiona McDougall, Eugenio M. Mercuri, Ivana Audhya, the EMBARK Study Group

Subjects: Get full text

A Korean Patient With Leber Congenital Amaurosis and a Homozygous RPE65 Variant Originating From a Paternal Uniparental Isodisomy by Hane Lee, Dongseok Moon, Rin Khang, Go Hun Seo, Chang Ki Yoon, Un Chul Park, Kyu Hyung Park, Eun Kyoung Lee

Subjects: Get full text

CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production by Emily E. O’Driscoll, Sakshi Arora, Jonathan F. Lang, Beverly L. Davidson, Ophir Shalem

Subjects: Get full text

Development of the Dutch translational knowledge agenda for inherited metabolic diseases by I. J. Hieltjes, J. H. van derLee, M. C. Groenendijk, G. vanHaaften, P. M. vanHasselt, R. J. Lunsing, G. J. J. vanProoijen, E. M. deRuiter, F. J. vanSpronsen, N. M. Verhoeven‐Duif, A. deVreugd, M. Wagenmakers, H. Zweers, H. Dekker, H. R. Waterham, C. D. vanKarnebeek, R. J. A. Wanders, R. A. Wevers

Subjects: “…gene therapy…”
Get full text

mTOR downregulation promotes anti-inflammatory responses via the CCL3-CCR5 axis in hypoxic retinopathy by Tae Kwon Moon, Im Kyeung Kang, Kyoung Jin Lee, Ji Hyun Kim, Hee Jong Kim, A. Reum Han, Ha-Na Woo, Joo Yong Lee, Jun-Sub Choi, Keerang Park, Heuiran Lee

Subjects: Get full text

Emerging Strategies in Cartilage Repair and Joint Preservation by Mircea Adrian Focsa, Sorin Florescu, Armand Gogulescu

Subjects: Get full text

Neuromuscular diseases: genomics-driven advances by Anna Cho

Subjects: Get full text

Cardiac Gene Therapy With Phosphodiesterase 2A Limits Remodeling and Arrhythmias in Mouse Models of Heart Failure by Rima Kamel, Aurélia Bourcier, Jean Piero Margaria, Valentin Jin, Audrey Varin, Agnès Hivonnait, Françoise Mercier‐Nomé, Delphine Mika, Alessandra Ghigo, Flavien Charpentier, Vincent Algalarrondo, Emilio Hirsch, Rodolphe Fischmeister, Grégoire Vandecasteele, Jérôme Leroy

Subjects: Get full text

Progress in photoreceptor replacement therapy for retinal degenerative diseases by Yuxin Du, Yin Shen

Subjects: Get full text

Efficient Dual Cas9 Nickase Correction of a Prevalent Pathogenic LAMB3 Variant for Junctional Epidermolysis Bullosa by Alex du Rand, John Hunt, Daniel Verdon, Ben Buttle, P. Rod Dunbar, Diana Purvis, Vaughan Feisst, Hilary Sheppard

Subjects: Get full text

Glutamine missense suppressor transfer RNAs inhibit polyglutamine aggregation by Rasangi Tennakoon, Teija M.I. Bily, Farah Hasan, Sunidhi Syal, Aaron Voigt, Tugce B. Balci, Kyle S. Hoffman, Patrick O’Donoghue

Subjects: Get full text

Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) a... by Wuh-Liang Hwu, Hui-Min Lee, John Devin Peipert, Rongrong Zhang, Christian Werner, J. Rafael Sierra, Thomas O’Connell, Jonathan J. Woolley, Marjorie Crowell, Antonia Wang, Ioannis Tomazos

Subjects: Get full text

The Genetic Basis of Congenital Cataracts: Advances in Diagnostics and Therapeutics by Agnieszka Starzyk, Piotr Charzewski

Subjects: Get full text

Silencing hepatic PCSK9 via novel chimeric AAV8 mitigates the progression of atherosclerosis by inhibiting inflammation in ApoE−/− mice by Xiaocui Chen, Minghui Sun, Xiang Ma, Yitong Ma, Bangdang Chen

Subjects: Get full text

Therapeutic landscape for inherited ocular diseases: current and emerging therapies by Hwei Wuen Chan, Jaslyn Oh, Bart Leroy

Subjects: Get full text