Results 51 to 60 of about 2,056 (145)

Anion‐Exchange Membrane Chromatography for the Separation of Empty and Full Adeno‐Associated Viral Capsids

Biotechnology Journal, Volume 21, Issue 8, August 2026.
Graphical Abstract and Lay Summary The anion‐exchange membrane elution chromatography for the separation of empty and full AAVs is explored and optimized. The role played by important parameters like flowrate, gradient slope, membrane loading, and feed residence time is studied in detail.
Luca Ossi, Samuele Delfino, Helena Marie, Michael Schulte, Mattia Sponchioni   +4 more
wiley   +1 more source

Variation in Health Technology Assessment recommendations and reimbursement of Advanced Therapy Medicinal Products across nine european countries

Discover Health Systems
Background Advanced Treatment Medicinal Products (ATMPs) are innovative treatments aimed at severe diseases. However, patient access varies across European countries. The present research reviews the reimbursement status of the licensed ATMPs in England,
Paraskevi M. Georgiou, Kostas P. Athanasakis   +1 more
doaj   +1 more source

Capture of adeno‐associated viruses from clarified lysate by continuous flow ultracentrifugation: a comparative study of iodixanol and sucrose gradients

Journal of Chemical Technology &Biotechnology, Volume 101, Issue 7, Page 1384-1395, July 2026.
Abstract BACKGROUND Adeno‐Associated Virus vectors are widely used in gene therapy due to their safety and efficacy. Despite the interest in their therapeutical application, AAV purification remains a significant challenge. Density gradient ultracentrifugation using cesium chloride, sucrose, or iodixanol gradients are common methods for AAV ...
Sabrina Leigheb, Maral Rahimzadeh, Nikolaus Virgolini, Aida Tleuova, Mahbod Mousavian, Astrid Dürauer, Patricia Pereira Aguilar   +6 more
wiley   +1 more source

Nuevo horizonte en el tratamiento de la Distrofia Hereditaria de la Retina.Terapia génica: Luxturna

, 2022
Aparicio Merinero, Carlos; director de proyecto: Tinoco Pastor, María Pilar2021-2022Grado en FarmaciaFacultad de Ciencias de la ...
Aparicio Merinero, Carlos
core   +1 more source

Reflections on emerging therapies for inherited retinal diseases following a community education event: A qualitative exploration

Optometry and Vision Science, Volume 103, Issue 7, July 2026.
ABSTRACT Purpose To explore the inherited retinal diseases (IRD) communities' attitudes and perspective toward emerging therapies—specifically gene therapy, cell therapy, and retinal prostheses—following a community education event. Methods At an in‐person half‐day IRD community education event, subject‐matter experts presented the scientific ...
Eden G. Robertson, Jorden Hua, Kate Hetherington, Michelle O’Hara‐Wright, Anai Gonzalez‐Cordero   +4 more
wiley   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, Volume 123, Issue 5, Page 1107-1122, May 2026.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source

Review of gene therapies for age-related macular degeneration [PDF]

, 2022
Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management.
Thomas, Mathew J, Kiss, Szilárd, Kaiser, Peter K, Waheed, Nadia K, Thomas, Mathew J., Weng, Christina Y., Yiu, Glenn, Kaiser, Peter K., Aziz, Aamir A, Aziz, Aamir A., Danzig, Carl J., Danzig, Carl J, Khanani, Arshad M., Khanani, Arshad M, Weng, Christina Y, Waheed, Nadia K.   +15 more
core   +1 more source

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, Volume 38, Issue 22, 17 April 2026.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

Terapia gênica in vivo e ex vivo: análise das tecnologias aplicadas em medicamentos aprovados pela Anvisa [PDF]

A terapia gênica, conceituada em 1972 por Theodore Friedmann e Richard Roblin, é um campo emergente que envolve técnicas de substituição, melhoria ou remodelação de genes para corrigir defeitos genéticos. A primeira aplicação bem-sucedida ocorreu em 1990,
Costa, André Lima de Oliveira, Vargas, Letícia Ribas, Oliveira, Vinicius Bednarczuk de   +2 more
core   +1 more source

Gene therapy for retinal diseases: From genetics to treatment

Indian Journal of Ophthalmology
The gene therapy approach for retinal disorders has been considered largely over the last decade owing to the favorable outcomes of the US Food and Drug Administration-approved commercial gene therapy, Luxturna.
Ashish Khaparde, Grace P Mathias, B Poornachandra, M B Thirumalesh, Rohit Shetty, Arkasubhra Ghosh   +5 more
doaj   +1 more source