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Sialic Acid-Engineered IL4–10 Fusion Protein is Bioactiveand Rapidly Cleared from the Circulation

Purpose Modulating sialylation of therapeutic glycoproteinsmay be used to influence their clearance and systemic exposure.We studied the effect of low and high sialylated IL4–10fusion protein (IL4–10 FP) on in vitro and in vivo bioactivity...
Authors: Cristine Steen-Louws & Peter Boross1 & Judith Prado & Jan Meeldijk1 & Jurgen B. Langenhorst &Alwin...

Virological Assessment of Hospitalized Patients With COVID-2019 

Coronavirus disease 2019 (COVID-19) is an acute respiratory tract infection that emerged in late 20191,2. Initial outbreaks in China involved 13.8% cases with severe, and 6.1% with critical courses3. This severe presentation corresponds to the usage...
Authors: Roman Wölfel  1 , Victor M Corman  2 , Wolfgang...
SST Peer Reviewed

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome

Precise correction of the CD40LG gene in T cells and hematopoietic stem/progenitor cells (HSPC) holds promise for treating X-linked hyper-IgM Syndrome (HIGM1), but its actual therapeutic potential remains elusive. Here, we developed a...
Authors: Valentina Vavassori , Elisabetta Mercuri , Genni E Marcovecchio , Maria C Castiello , Giulia...

Candidate Markers for Stratification and Classification in Rheumatoid Arthritis

Rheumatoid arthritis (RA) is a chronic autoimmune, inflammatory disease, characterizedby synovitis in small- and medium-sized joints and, if not treated early and efficiently, jointdamage, and destruction. RA is a heterogeneous disease with a...
Authors: Lucius Bader, Stein-Erik Gullaksen, Nello Blaser, Morten Brun,Gerd Haga Bringeland, André Sulen,...

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

Abstract There has been a resurgence in gene therapyefforts that is partly fueled by the identification andunderstanding of new gene delivery vectors. Adeno-associatedvirus (AAV) is a non-enveloped virus that can beengineered to deliver DNA to target...
Authors: Michael F. Naso, Brian Tomkowicz, William L. Perry III, William R. Strohl

Development of ß-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000 births annually worldwide. SCD is an autosomal recessive disease resulting from a single point mutation in codon six of the ß-globin gene (HBB). Ex vivo ß-globin gene...
Authors: Annalisa Lattanzi , Joab Camarena , Premanjali Lahiri , Helen Segal , Waracharee Srifa , Christopher...

In vitro and in vivo evaluation of discogenic cells, an investigational cell therapy for disc degeneration

BACKGROUND/CONTEXT: Disc degeneration (DD) is a significant driver of low back painand few treatments exist to treat the pain and disability associated with the disease.PURPOSE: Our group has developed a method to generate therapeutic discogenic...
Authors: Lara Ionescu Silverman, Galina Dulatova,Terry Tandeski, Isaac E. Erickson,  Beverly Lundell, David...

Perspectives on monoclonal antibody therapy as potential therapeutic intervention for Coronavirus disease-19 (COVID-19)

Last decade witnessed the outbreak of many life-threatening human pathogens including Nipah, Ebola, Chikungunya, Zika, Middle East respiratory syndrome coronavirus (MERS-CoV), Severe Acute respiratory...
Authors: Shanmugaraj B1,2, Siriwattananon K1,2, Wangkanont K3, Phoolcharoen W1,2.
SST Peer Reviewed

Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression

Background Chimeric antigen receptor (CAR) T cells have demonstrated high clinical response rates against hematological malignancies (e.g., CD19+ cancers) buthave shown limited activity in patients with solid tumors. Recent work showed that precise...
Authors: Mueller KP, Piscopo NJ, Forsberg MH, Saraspe LA, Das A, Russell B, Smerchansky M, Cappabianca D, Shi...
SST Peer Reviewed

Sperm DNA damage causes genomic instability in early embryonic development

Genomic instability is common in human embryos, but the underlying causes are largely unknown. Here, weexamined the consequences of sperm DNA damage on the embryonic genome by single-cell whole-genomesequencing of individual blastomeres from bovine...
Authors: Sjors Middelkamp, Helena T. A. van Tol, Diana C. J. Spierings, Sander Boymans,Victor Guryev, Bernard...
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