Modifying enzyme replacement therapy – A perspective

Primary B cell engineering for therapeutic research

(2022) Evan Kleinboehl et al.   TRENDS IN MOLECULAR MEDICINE

Genetic engineering of T cells for immunotherapy

(2021) Gavin I. Ellis et al.   NATURE REVIEWS GENETICS

T cell circuits that sense antigen density with an ultrasensitive threshold

(2021) Rogelio A. Hernandez-Lopez et al.   SCIENCE

Haemophilia

(2021) Erik Berntorp et al.   Nature Reviews Disease Primers

Rapid Progression of Angioimmunoblastic T Cell Lymphoma Following BNT162b2 mRNA Vaccine Booster Shot: A Case Report

(2021) Serge Goldman et al.   Frontiers in Medicine

Emerging Approaches for Regulation and Control of CAR T Cells: A Mini Review

(2020) Lærke J. B. Brandt et al.   Frontiers in Immunology

Plasma proteomic profiling of young and old mice reveals cadherin-13 prevents age-related bone loss

(2020) Yong Ryoul Yang et al.   Aging-US

Targeting Strategies for Tissue-Specific Drug Delivery

(2020) Zongmin Zhao et al.   CELL

Dynamics in protein translation sustaining T cell preparedness

(2020) Tobias Wolf et al.   NATURE IMMUNOLOGY

Precise T cell recognition programs designed by transcriptionally linking multiple receptors

(2020) Jasper Z. Williams et al.   SCIENCE

Specific factors in blood from young but not old mice directly promote synapse formation and NMDA-receptor recruitment

(2019) Kathlyn J. Gan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

B cells engineered to express pathogen-specific antibodies protect against infection

(2019) Howell F. Moffett et al.   Science Immunology

In Vivo Translation of Peptide-Targeted Drug Delivery Systems Discovered by Phage Display

(2018) Maureen R. Newman et al.   BIOCONJUGATE CHEMISTRY

A guide to manufacturing CAR T cell therapies

(2018) Philipp Vormittag et al.   CURRENT OPINION IN BIOTECHNOLOGY

Engineering Protein-Secreting Plasma Cells by Homology-Directed Repair in Primary Human B Cells

(2018) King L. Hung et al.   MOLECULAR THERAPY

The exerkine apelin reverses age-associated sarcopenia

(2018) Claire Vinel et al.   NATURE MEDICINE

Growth Differentiation Factor 11 treatment leads to neuronal and vascular improvements in the hippocampus of aged mice

(2018) Ceren Ozek et al.   Scientific Reports

Lysosomal storage diseases

(2018) Frances M. Platt et al.   Nature Reviews Disease Primers

Enzyme replacement therapy and beyond—in memoriam Roscoe O. Brady, M.D. (1923–2016)

(2017) Markus Ries   JOURNAL OF INHERITED METABOLIC DISEASE

Human umbilical cord plasma proteins revitalize hippocampal function in aged mice

(2017) Joseph M. Castellano et al.   NATURE

Supraphysiologic control over HIV-1 replication mediated by CD8 T cells expressing a re-engineered CD4-based chimeric antigen receptor

(2017) Rachel S. Leibman et al.   PLoS Pathogens

Precision Tumor Recognition by T Cells With Combinatorial Antigen-Sensing Circuits

(2016) Kole T. Roybal et al.   CELL

Targeted antibody-mediated depletion of murine CD19 CAR T cells permanently reverses B cell aplasia

(2016) Paulina J. Paszkiewicz et al.   JOURNAL OF CLINICAL INVESTIGATION

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

β2-microglobulin is a systemic pro-aging factor that impairs cognitive function and neurogenesis

(2015) Lucas K Smith et al.   NATURE MEDICINE

Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome

(2015) Abhijit Ricky Pal et al.   Orphanet Journal of Rare Diseases

Relation of clinical culture method to T-cell memory status and efficacy in xenograft models of adoptive immunotherapy

(2014) David M. Barrett et al.   CYTOTHERAPY

OX40 Costimulatory Signals Potentiate the Memory Commitment of Effector CD8+ T Cells

(2014) S. F. Mousavi et al.   JOURNAL OF IMMUNOLOGY

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

(2014) Christian J. Hendriksz et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Young blood reverses age-related impairments in cognitive function and synaptic plasticity in mice

(2014) Saul A Villeda et al.   NATURE MEDICINE

Antigen affinity, costimulation, and cytokine inputs sum linearly to amplify T cell expansion

(2014) J. M. Marchingo et al.   SCIENCE

Vascular and Neurogenic Rejuvenation of the Aging Mouse Brain by Young Systemic Factors

(2014) L. Katsimpardi et al.   SCIENCE

Restoring Systemic GDF11 Levels Reverses Age-Related Dysfunction in Mouse Skeletal Muscle

(2014) M. Sinha et al.   SCIENCE

The inducible caspase-9 suicide gene system as a “safety switch” to limit on-target, off-tumor toxicities of chimeric antigen receptor T cells

(2014) Tessa Gargett et al.   Frontiers in Pharmacology

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors

(2013) H. Torikai et al.   BLOOD

Growth Differentiation Factor 11 Is a Circulating Factor that Reverses Age-Related Cardiac Hypertrophy

(2013) Francesco S. Loffredo et al.   CELL

Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study

(2013) M. J. Manco-Johnson et al.   HAEMOPHILIA

Mucopolysaccharidosis type VI phenotypes-genotypes and antibody response to galsulfase

(2013) Marion M Brands et al.   Orphanet Journal of Rare Diseases

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR

(2012) H. Torikai et al.   BLOOD

T-cell receptor diversity prevents T-cell lymphoma development

(2012) S Newrzela et al.   LEUKEMIA

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

(2012) Elena Provasi et al.   NATURE MEDICINE

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase therapy in the real-world setting: A perspective from the Hunter Outcome Survey (HOS)

(2011) Barbara K. Burton et al.   MOLECULAR GENETICS AND METABOLISM

The ageing systemic milieu negatively regulates neurogenesis and cognitive function

(2011) Saul A. Villeda et al.   NATURE

A human memory T cell subset with stem cell–like properties

(2011) Luca Gattinoni et al.   NATURE MEDICINE

T Cells with Chimeric Antigen Receptors Have Potent Antitumor Effects and Can Establish Memory in Patients with Advanced Leukemia

(2011) M. Kalos et al.   Science Translational Medicine

Computational design of peptide ligands

(2011) Peter Vanhee et al.   TRENDS IN BIOTECHNOLOGY

The PD-1 pathway in tolerance and autoimmunity

(2010) Loise M. Francisco et al.   IMMUNOLOGICAL REVIEWS

The immunogenicity of humanized and fully human antibodies

(2010) Fiona A. Harding et al.   mAbs

Retroviral transfer of human CD20 as a suicide gene for adoptive T-cell therapy

(2009) M. Griffioen et al.   HAEMATOLOGICA

Cyclical expression of L-selectin (CD62L) by recirculating T cells

(2009) A. Klinger et al.   INTERNATIONAL IMMUNOLOGY

Chimeric Receptors Containing CD137 Signal Transduction Domains Mediate Enhanced Survival of T Cells and Increased Antileukemic Efficacy In Vivo

(2009) Michael C. Milone et al.   MOLECULAR THERAPY

Intrinsic and extrinsic control of effector T cell survival and memory T cell development

(2008) Timothy W. Hand et al.   IMMUNOLOGIC RESEARCH

Effects of IL-7 on memory CD8+ T cell homeostasis are influenced by the timing of therapy in mice

(2008) Som G. Nanjappa et al.   JOURNAL OF CLINICAL INVESTIGATION

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

(2008) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

(2008) L. A. Clarke et al.   PEDIATRICS