Reversible Cysteine Acylation Regulates the Activity of Human Palmitoyl-Protein Thioesterase 1 (PPT1)

Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease)

(2013) Jacqueline A. Hawkins-Salsbury et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Treatment of the Ppt1–/– Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis With the N-methyl-d-aspartate (NMDA) Receptor Antagonist Memantine

(2013) Rozzy Finn et al.   JOURNAL OF CHILD NEUROLOGY

Deterministic direct reprogramming of somatic cells to pluripotency

(2013) Yoach Rais et al.   NATURE

Neuroprotection and lifespan extension in Ppt1 −/− mice by NtBuHA: therapeutic implications for INCL

(2013) Chinmoy Sarkar et al.   NATURE NEUROSCIENCE

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

(2012) Shannon L. Macauley et al.   ANNALS OF NEUROLOGY

Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

(2012) Marie S. Roberts et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

(2012) Jie Hu et al.   MOLECULAR GENETICS AND METABOLISM

Therapeutic Approaches to the Challenge of Neuronal Ceroid Lipofuscinoses

(2011) R. Kohan et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Subcellular Golgi localization of stathmin family proteins is promoted by a specific set of DHHC palmitoyl transferases

(2011) Aurore D. Levy et al.   MOLECULAR BIOLOGY OF THE CELL

FKBP12 Binds to Acylated H-Ras and Promotes Depalmitoylation

(2011) Ian M. Ahearn et al.   MOLECULAR CELL

Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients

(2011) Chinmoy Sarkar et al.   MOLECULAR GENETICS AND METABOLISM

Palmitoyl:protein thioesterase (PPT1) inhibitors can act as pharmacological chaperones in infantile Batten disease

(2010) Glyn Dawson et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Derivation of Pre-X Inactivation Human Embryonic Stem Cells under Physiological Oxygen Concentrations

(2010) Christopher J. Lengner et al.   CELL

Protein palmitoylation in neuronal development and synaptic plasticity

(2010) Yuko Fukata et al.   NATURE REVIEWS NEUROSCIENCE

Towards understanding the neuronal ceroid lipofuscinoses

(2009) Alfried Kohlschütter et al.   BRAIN & DEVELOPMENT

Ndel1 palmitoylation: a new mean to regulate cytoplasmic dynein activity

(2009) Anat Shmueli et al.   EMBO JOURNAL

Neuronal palmitoyl acyl transferases exhibit distinct substrate specificity

(2009) Kun Huang et al.   FASEB JOURNAL

Mobile DHHC palmitoylating enzyme mediates activity-sensitive synaptic targeting of PSD-95

(2009) Jun Noritake et al.   JOURNAL OF CELL BIOLOGY

Robust Fluorescent Detection of Protein Fatty-Acylation with Chemical Reporters

(2009) Guillaume Charron et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis

(2009) Jui-Yun Lu et al.   MOLECULAR GENETICS AND METABOLISM

Large-scale profiling of protein palmitoylation in mammalian cells

(2009) Brent R Martin et al.   NATURE METHODS

Neuronal ceroid lipofuscinoses

(2008) Anu Jalanko et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Palmitoyl protein thioesterase-1 deficiency impairs synaptic vesicle recycling at nerve terminals, contributing to neuropathology in humans and mice

(2008) Sung-Jo Kim et al.   JOURNAL OF CLINICAL INVESTIGATION

Neural palmitoyl-proteomics reveals dynamic synaptic palmitoylation

(2008) Rujun Kang et al.   NATURE

CSS-Palm 2.0: an updated software for palmitoylation sites prediction

(2008) J. Ren et al.   PROTEIN ENGINEERING DESIGN & SELECTION