Results 81 to 90 of about 45,012 (247)

Astatine‐211—Towards In Vivo Stable Astatine‐211 Labeled Radiopharmaceuticals and Their (Pre)Clinical Applications

Medicinal Research Reviews, EarlyView.
ABSTRACT Targeted radioligand therapy has emerged as a promising treatment option for eradicating advanced cancer forms. α‐Emitters are considered particularly promising as they can obliterate (micro)‐metastases. The α‐emitter astatine‐211 (211At) has experienced increased interest due to its favorable decay properties.
Marius Müller, Nadia Bom Pedersen, Vladimir Shalgunov, Andreas Ingemann Jensen, Umberto Maria Battisti, Matthias Manfred Herth   +5 more
wiley   +1 more source

Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency [PDF]

, 2014
Gene transfer into autologous hematopoietic stem cells by γ-retroviral vectors (gRV) is an effective treatment for adenosine deaminase (ADA)–deficient severe combined immunodeficiency (SCID).
Blundell, Michael P., Carbonaro, Denise A., Carmo, Marlene, Chan, Rebecca Y., Cooper, Aaron, Cornetta, Kenneth, Fairbanks, Lynette, Fu, Pei-Yu, Gaspar, H. Bobby, Geiger, Sabine, Hollis, Roger P., Jin, Xiangyang, Kaufman, Michael L., Kohn, Donald B., Montiel-Equihua, Claudia, Sahaghian, Arineh, Sebire, Neil J., Senadheera, Shantha, Thrasher, Adrian J., Wang, Xiaoyan, Zhang, Lin   +20 more
core   +1 more source

An Integrated Method to Evaluate the Treatment Efficacy and Immunogenicity of B‐Cell Acute Lymphoblastic Leukemia Therapeutic Enzyme Formulations

Small Methods, EarlyView.
This work presents an integrated set of methods to evaluate the efficacy of asparaginase formulations against B‐cell Acute Lymphoblastic Leukemia (ALL) in mice, including the establishment of an ALL murine model, an amino acid depletion study using high‐performance liquid chromatography (HPLC), and comprehensive assessment of antibody production ...
Julien Santelli, Annie Y. Heble, Connor Endsley, Adam Woodward, Nazia Hafeez, Naomi Winick, Jacques Lux   +6 more
wiley   +1 more source

Intrathymic injection of hematopoietic progenitor cells establishes functional T cell development in a mouse model of severe combined immunodeficiency

Journal of Hematology & Oncology, 2017
Background Even though hematopoietic stem cell transplantation can be curative in patients with severe combined immunodeficiency, there is a need for additional strategies boosting T cell immunity in individuals suffering from genetic disorders of ...
Andrea Z. Tuckett, Raymond H. Thornton, Richard J. O’Reilly, Marcel R. M. van den Brink, Johannes L. Zakrzewski   +4 more
doaj   +1 more source

Congenital microcephaly [PDF]

, 2014
The underlying etiologies of genetic congenital microcephaly are complex and multifactorial. Recently, with the exponential growth in the identification and characterization of novel genetic causes of congenital microcephaly, there has been a ...
Agha, Ahmad, Arai, Banin, Bettencourt-Dias, Bicknell, Bicknell, Bolanos-Garcia, Bond, Bongers, Buchman, Caldecott, Caldecott, Capo-Chichi, Carney, Casey, Cimprich, Cizmecioglu, Croteau, Dehay, Dehay, Deriano, Domchek, Fish, Gatz, Gellert, Genin, Gilmore, Gineau, Gorlin, Griffith, Gruber, Guernsey, Han, Hanks, Heldin, Hemerly, Herzog, Heyer, Hickson, Hinchcliffe, Hughes, Issa, Jaglin, Jamieson, Kang, Kee, Kerzendorfer, Kim, Kiyomitsu, Kiyomitsu, Koch, Kumagai, Kuo, Lancaster, Lavin, Lee, Lee, Lee, Lobrich, Loveday, Loveday, Mahmood, Maréchal, Matsumoto, Matsuoka, Matsuura, Meindl, Mimitou, Mirzaa, Miyoshi, Miyoshi, Moldovan, Morris-Rosendahl, Moshous, Moynahan, Murga, Murray, Musacchio, Mühlhans, Noguchi, Novorol, O'Driscoll, O'Driscoll, O'Driscoll, O'Driscoll, O'Driscoll, O'Driscoll, Ogi, Orii, Pilaz, Poirier, Ponting, Qvist, Rakic, Rauch, Riballo, Romaniello, Roos, Rudner, Schneider, Shah, Shaheen, Shaheen, Shamseldin, Shen, Shiloh, Sir, Sirbu, Sokka, Spalding, Spassky, Stewart, Stiff, Stokes, Symington, Tan, Thornton, van der Burg, van der Lelij, Varon, Verloes, Walczak, Waltes, Weedon, Weinfeld, Woodbine, Woods, Wu, Zimmerman   +129 more
core   +1 more source

UK recommendations for chimerism testing and monitoring following allogeneic haematopoietic stem cell transplantation (HSCT): Best practice consensus guidelines from the British Society for Blood and Marrow Transplant and Cellular Therapies (BSBMTCT), NHS England Genomic Laboratory Hub (GLH) Haematological Malignancies Working Group, UK Cancer Genetics Group (UKCGG) and the UK National External Quality Assessment Service for Leucocyte Immunophenotyping (UK NEQAS LI)

British Journal of Haematology, EarlyView.
Summary In allogeneic haematopoietic stem cell transplantation (HSCT), important clinical decisions depend upon assessment of chimerism, including immunosuppressant dosing and donor lymphocyte infusions (DLI), which in turn can have major impacts on disease control, graft‐versus‐host disease (GVHD), immunity and ultimately patient survival.
Andrew Clark, Hazel Clouston, Kanchan Rao, Najeem Folarin, Josu De la Fuente, Angela Hamblin, Eduardo Olavarria, Debbie Richardson, Polly Talley, Victoria Potter, Justin Loke, Terri McVeigh, John Snowden   +12 more
wiley   +1 more source

Development of a Synthetic Lethality‐Based Combination Therapy Using LIG1 and PARP Inhibitors for Prostate Cancer

Cancer Science, EarlyView.
This study identified LIG1 as a novel synthetic lethality‐inducing factor in prostate cancer through a genome‐wide CRISPR‐Cas9 knockout screen and demonstrated that its inhibition enhances the efficacy of PARP inhibitor. The combination of L82‐G17 and olaparib showed synergistic antitumor effects both in vitro and in vivo, offering a promising ...
Masaru Tani, Koji Hatano, Yu Ishizuya, Toshiki Oka, Tomohiro Kanaki, Shunsuke Inoguchi, Akihiro Yoshimura, Yuki Horibe, Yutong Liu, Sassi Nesrine, Yohei Okuda, Akinaru Yamamoto, Toshihiro Uemura, Gaku Yamamichi, Takuji Hayashi, Yoshiyuki Yamamoto, Taigo Kato, Atsunari Kawashima, Takao Yamaguchi, Satoshi Obika, Kosuke Yusa, Norio Nonomura, Keisuke Nimura   +22 more
wiley   +1 more source

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo [PDF]

, 2007
We treated 10 children with X-linked SCID (SCID-X1) using gammaretrovirus-mediated gene transfer. Those with sufficient follow-up were found to have recovered substantial immunity in the absence of any serious adverse events up to 5 years after treatment.
Kerstin Schwarzwaelder, Steven J. Howe, Manfred Schmidt, Martijn H. Brugman, Annette Deichmann, Hanno Glimm, Sonja Schmidt, Claudia Prinz, Manuela Wissler, Douglas J.S. King, Fang Zhang, Kathryn L. Parsley, Kimberly C. Gilmour, Joanna Sinclair, Jinhua Bayford, Rachel Peraj, Karin Pike-Overzet, Frank J.T. Staal, Dick de Ridder, Christine Kinnon, Ulrich Abel, Gerard Wagemaker, H. Bobby Gaspar, Adrian J. Thrasher, Christof von Kalle, Cavazzana-Calvo, Gaspar, Hacein-Bey-Abina, Aiuti, Aiuti, Gaspar, Ott, Hacein-Bey-Abina, Li, Kustikova, Modlich, Montini, Mitchell, Schröder, Wu, Bushman, Schmidt, Schmidt, Hematti, Laufs, Suzuki, Bartholomew, Recchia, Calmels, Hoeben, Palmer, Xu, Dik, Ge   +53 more
core   +2 more sources

Making Advanced Therapies Affordable and Accessible: Two Strategic Approaches

Developing World Bioethics, EarlyView.
ABSTRACT This article explores two complementary strategies for addressing the affordability and access challenges facing advanced therapies. As high development costs and limited market access have led to the withdrawal of several therapies, the article examines how these barriers create ‘valleys of death’ that prevent innovation from reaching ...
Ubaka Ogbogu, Lauren Albrecht
wiley   +1 more source

Successful Handling of Disseminated BCG Disease in a Child with Severe Combined Immunodeficiency

Case Reports in Medicine, 2011
In high-burden countries, Mycobacterium bovis Bacillus Calmette-Guérin (BCG) vaccine is administered in newborn to prevent severe Mycobacterium tuberculosis infection.
Sílvia Bacalhau, Cristina Freitas, Rosalina Valente, Deolinda Barata, Conceição Neves, Katrin Schäfer, Annelie Lubatschofski, Ansgar Schulz, João Farela Neves   +8 more
doaj   +1 more source