Results 191 to 200 of about 65,616 (261)

Technical and biological sources of noise confound multiplexed enhancer AAV screening. [PDF]

Nat Commun
Hunker AC, Mich JK, Taskin N, Torkelson A, Cardenas T, Lalanne JB, Mahoney JT, Bertagnolli D, Chakka AB, Chakrabarty R, Donadio NP, Ferrer R, Gasperini M, Goldy J, Guzman J, Jin K, Khem S, Kutsal R, Martinez RA, Newman D, Pena N, Rimorin C, Rocha D, Shapovalova NV, Tieu M, Weed N, Zhou T, Hodge R, Yao S, Shendure J, Smith KA, Lein ES, Tasic B, Levi BP, Ting JT.   +34 more
europepmc   +1 more source

Demonstration of nanofiltration‐based virus safety for AAV manufacturing using model viruses

Biotechnology Progress, EarlyView.
Abstract Virus safety for biopharmaceutical products has been a well‐established concern for several decades. While various virus reduction steps have been identified and implemented for traditional plasma‐derived and monoclonal antibody products, there are various challenges to achieving the same virus safety in emerging gene therapy products.
Brian Buesing, Tomoki Igi, Daniel Strauss   +2 more
wiley   +1 more source

A female-specific role for Slit1 in prenatal stress-induced depression vulnerability. [PDF]

Neurotherapeutics
Xu K, Chen T, Yang Y, Liu W, Guo H, Gong Z, Ma X, Liu J, Lu Y, Zhang H, Fan X, Li H, Zhu Z.   +12 more
europepmc   +1 more source

High‐throughput development and scale‐up of recombinant adeno‐associated virus 5 anion‐exchange chromatography for high‐purity capsid separation

Biotechnology Progress, EarlyView.
High‐throughput anion‐exchange chromatography process development enables scalable purification of high‐purity recombinant adeno‐associated virus capsids. Abstract Recombinant adeno‐associated virus (rAAV) production commonly yields a high proportion of empty and partially filled capsids, necessitating downstream purification to enrich full capsids ...
Kevin Vera, Ivan Gajic, Praveen Kusumanchi, Angela Andaluz, Gabrielle Dela Pena, Clifford A. Froelich, Elizabeth Higgins, Jennifer Johnston, Seyed Pouria Motevalian   +8 more
wiley   +1 more source

AAV2-associated liver injury across infection and gene therapy: converging genomic signals. [PDF]

Front Immunol
Chen Y, Zhu Q, Yuan N, Jin D, Zhu Z, Chen F.   +5 more
europepmc   +1 more source

Cytoskeleton–Membrane Uncoupling in Duchenne Muscular Dystrophy: Implications for Newborn Screening and Early Protection

Cytoskeleton, EarlyView.
ABSTRACT The cytoskeleton of striated muscle integrates force transmission, mechanotransduction, and sarcolemmal stability through coordinated networks of sarcomeres, costameres, and intermediate filaments. Together, these systems establish mechanical continuity between the contractile apparatus, the sarcolemma, and the extracellular matrix.
Houda Cohen, Nora Hosny, Brian R. Thompson, Joseph M. Metzger   +3 more
wiley   +1 more source

Downregulating <i>Nrl</i> Expression and Rod Photoreceptor Protection. [PDF]

Int J Mol Sci
Li Y, Jiao S, Tao W, Wen R.
europepmc   +1 more source

AAV Vectors

Molecular Therapy, 2001
openaire   +1 more source

Clinical Model‐Informed Precision Dosing Consult Service for Accelerating Personalized Medication in Pediatric Patients

Clinical Pharmacology &Therapeutics, EarlyView.
Traditional dosing strategies often rely on a “one‐size‐fits‐all” paradigm, assuming an “average” patient with typical demographic and pharmacological characteristics. In reality, this often overlooks existing between‐patient variability and can lead to suboptimal drug exposure or toxicity. This issue is especially pronounced in pediatric patients, who
Zachary L. Taylor, Takanobu Nadai, Kei Irie, Wen Rui Tan, Sanjana Parikh, Anhar Hosawi, Matthijs W. van Hoogdalem, Ronaldo Morales Junior, Sonya Tang Girdwood, Abbie D. Leino, Kana Mizuno, Alexander A. Vinks, Tomoyuki Mizuno   +12 more
wiley   +1 more source

Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer. [PDF]

Mol Ther
Xicluna R, Yamada K, Gonzalez-Visiedos M, Greenwood CM, Zaugg D, Waiz D, Steiner G, Hahn K, Schwandt T, Otteneder MB, Biswas M, Muñoz-Melero M, Kumar SRP, Herzog RW, Haegel H.   +14 more
europepmc   +1 more source