Results 31 to 40 of about 6,300,245 (266)

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

EMBO Molecular Medicine
Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), however, adverse ...
Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao, Jun Xie   +16 more
doaj   +1 more source

Classical Complement Pathway Inhibition in a Pediatric Patient With Refractory Immune Thrombocytopenia

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Pediatric refractory immune thrombocytopenia (rITP) is generally characterized by persistent thrombocytopenia and failure to achieve a sustained response to multiple standard therapies. We report a 4‐year‐old female with congenital tufting enteropathy, post‐intestinal transplant patient on immunosuppressants, who developed severe rITP ...
Sarah Hsieh, Susmita Sarangi, Caileigh Pudela, Catherine M. Broome, Tara K. Suntum   +4 more
wiley   +1 more source

Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India

, 2022
Gene and cell therapies for a variety of life-limiting illnesses are under investigation, and a small number of commercial products have successfully obtained regulatory approval.
Bonamino, Martín, Mingozzi, Federico, Cornetta, Kenneth, Rangarajan, Savita, Mahlangu, Johnny, Rao, Jayandharan, Martín Bonamino, Johnny Mahlangu, Federico Mingozzi, Jayandharan Rao, Savita Rangarajan, Kenneth Cornetta   +11 more
core   +1 more source

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Molecular Therapy: Methods & Clinical Development, 2018
The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To date,
Shelley A. Nass, Maryellen A. Mattingly, Denise A. Woodcock, Brenda L. Burnham, Jeffrey A. Ardinger, Shayla E. Osmond, Amy M. Frederick, Abraham Scaria, Seng H. Cheng, Catherine R. O’Riordan   +9 more
doaj   +1 more source

Idiotype vaccines produced with a non-cytopathic alphavirus self-amplifying RNA vector induce antitumor responses in a murine model of B-cell lymphoma

Scientific Reports, 2021
A promising therapy for patients with B-cell lymphoma is based on vaccination with idiotype monoclonal antibodies (mAbs). Since idiotypes are different in each tumor, a personalized vaccine has to be produced for each patient.
Erkuden Casales, Eva Martisova, Helena Villanueva, Ascensión López Díaz de Cerio, Susana Inoges, Noelia Silva-Pilipich, María Cristina Ballesteros-Briones, Alejandro Aranda, Jaione Bezunartea, Maurizio Bendandi, Fernando Pastor, Cristian Smerdou   +11 more
doaj   +1 more source

Bone and Soft Tissue Neoplasms in Infants Versus Older Children: A Report From the Cancer in Young People in Canada Program

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background and Aims Infant bone and soft tissue neoplasms present distinct clinical challenges and lack large‐scale data to inform treatment decisions. This study aims to characterize bone and soft tissue neoplasms in infants in Canada and to compare their outcomes with those of older pediatric patients.
Samuel Sassine, Hallie Coltin, Maria Kondyli, Monia Marzouki, Nicolas Prud'homme, Nida Usmani, Rebecca J. Deyell, Lesleigh Abbott, Tony H. Truong, Sapna Oberoi, Ketan Kulkarni, Josée Brossard, Lynette Bowes, Paul Gibson, Sarah McKillop, Roona Sinha, Lillian Sung, Catherine Vezina, Laura Wheaton, Alexandra P. Zorzi, Marie‐Claude Pelland‐Marcotte, Thai Hoa Tran   +21 more
wiley   +1 more source

Neurocognitive, School‐Based, and Quality of Life Outcomes for Youth With Sickle Cell Disease and Thalassemia: A Scoping Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT The medical burdens of sickle cell disease (SCD) and transfusion‐dependent β‐thalassemia (TDT) are chronic and cumulative, even in children. Yet the effects expand beyond medications and healthcare visits due to their impacts on school attendance, academic performance, and peer engagement.
Mary Ann Etling, Hakim Tokhi, Alexander A. Boucher   +2 more
wiley   +1 more source

Progress and prospects: gene therapy clinical trials (part 1)

, 2012
Over the last two decades gene therapy has moved from preclinical to clinical studies for many diseases ranging from single gene disorders such as cystic fibrosis and Duchenne muscular dystrophy, to more complex diseases such as cancer and cardiovascular
Simons, M., Bainbridge, J. W., Yla-Herttuala, S., Alton, E.W.F., Kaplitt, M. G., Alexander, B. L., Ali, R. R., Braun, S., Grez, M., Cheng, S. H., Seger, R., Flotte, Terence R., Ott, M. G., Gaspar, H. B., Griesenbach, U., Thrasher, A. Z.   +15 more
core   +1 more source

Real-time MR tracking of AAV gene therapy with betagal-responsive MR probe in a murine model of GM1-gangliosidosis [PDF]

, 2021
Full author list omitted for brevity. For the full list of authors, see article.Transformative results of adeno-associated virus (AAV) gene therapy in patients with spinal muscular atrophy and Leber's congenital amaurosis led to approval of the first two
Gray-Edwards, Heather L, Rodriguez, Paola, Sena-Esteves, Miguel, Taghian, Toloo, Todeasa, Sophia H., Meade, Thomas J., King, Robert M., Gounis, Matthew J, Batista, Ana Rita, Zheng, Shaokuan, Mesa, Katerina   +10 more
core   +1 more source

Establishing the cost of implementing a performance-based, managed entry agreement for a hypothetical CAR T-cell therapy

Journal of Market Access & Health Policy, 2018
Background: Market access stakeholders consider the adoption of Managed Entry Agreements (MEAs), however a clearly described methodology to quantify their implementation burden is not available in the public domain.
Panos Kefalas, Omar Ali, Jesper Jørgensen, Nick Merryfield, Tim Richardson, Adam Meads, Laura Mungapen, Matthew Durdy   +7 more
doaj   +1 more source