A bacterial glycosidase enables mannose-6-phosphate modification and improved cellular uptake of yeast-produced recombinant human lysosomal enzymes.

Abstract:

:Lysosomal storage diseases are treated with human lysosomal enzymes produced in mammalian cells. Such enzyme therapeutics contain relatively low levels of mannose-6-phosphate, which is required to target them to the lysosomes of patient cells. Here we describe a method for increasing mannose-6-phosphate modification of lysosomal enzymes produced in yeast. We identified a glycosidase from C. cellulans that 'uncaps' N-glycans modified by yeast-type mannose-Pi-6-mannose to generate mammalian-type N-glycans with a mannose-6-phosphate substitution. Determination of the crystal structure of this glycosidase provided insight into its substrate specificity. We used this uncapping enzyme together with α-mannosidase to produce in yeast a form of the Pompe disease enzyme α-glucosidase rich in mannose-6-phosphate. Compared with the currently used therapeutic version, this form of α-glucosidase was more efficiently taken up by fibroblasts from Pompe disease patients, and it more effectively reduced cardiac muscular glycogen storage in a mouse model of the disease.

journal_name

Nat Biotechnol

journal_title

Nature biotechnology

authors

Tiels P,Baranova E,Piens K,De Visscher C,Pynaert G,Nerinckx W,Stout J,Fudalej F,Hulpiau P,Tännler S,Geysens S,Van Hecke A,Valevska A,Vervecken W,Remaut H,Callewaert N

doi

10.1038/nbt.2427

subject

Has Abstract

pub_date

2012-12-01 00:00:00

pages

1225-31

issue

12

eissn

1087-0156

issn

1546-1696

pii

nbt.2427

journal_volume

30

pub_type

杂志文章
  • Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

    abstract::Genome editing with targeted nucleases and DNA donor templates homologous to the break site has proven challenging in human hematopoietic stem and progenitor cells (HSPCs), and particularly in the most primitive, long-term repopulating cell population. Here we report that combining electroporation of zinc finger nucle...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.3408

    authors: Wang J,Exline CM,DeClercq JJ,Llewellyn GN,Hayward SB,Li PW,Shivak DA,Surosky RT,Gregory PD,Holmes MC,Cannon PM

    更新日期:2015-12-01 00:00:00

  • Antibodies engineered with IgD specificity efficiently deliver integrated T-cell epitopes for antigen presentation by B cells.

    abstract::We have developed a strategy for improving the stimulation of T cells during immune responses by constructing recombinant antibodies that enhance the delivery of antigen to antigen-presenting cells, such as B cells. These antibodies have variable regions specific for surface molecules on B cells, and a constant region...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/10883

    authors: Lunde E,Munthe LA,Vabø A,Sandlie I,Bogen B

    更新日期:1999-07-01 00:00:00

  • Almost in bloom.

    abstract::St. Louis wants to become a hub of agricultural biotechnology. All it needs is more start-ups and funds. ...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt0408-471

    authors: Marris E

    更新日期:2008-04-01 00:00:00

  • A signal sequence trap based on a constitutively active cytokine receptor.

    abstract::Targeting of secreted and cell-surface proteins to the cell membrane is mediated by a short hydrophobic stretch of amino acids, termed the signal sequence. We have developed a method that detects signal sequences in cDNA fragments based on their ability to redirect a constitutively active mutant of a cytokine receptor...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/8666

    authors: Kojima T,Kitamura T

    更新日期:1999-05-01 00:00:00

  • Efficient hammerhead ribozyme and antisense RNA targeting in a slow ribosome Escherichia coli mutant.

    abstract::We have evaluated inhibition of the plasmid-born chloramphenicol acetyl transferase gene (CAT) by the hammerhead ribozyme and antisense RNA in Escherichia coli where the translation and transcription rates have been modified. Whereas neither antisense nor the hammerhead had an inhibitory effect on CAT activity in wild...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt0597-432

    authors: Chen H,Ferbeyre G,Cedergren R

    更新日期:1997-05-01 00:00:00

  • Blockade of CD2-LFA-3 interactions protects human skin allografts in immunodeficient mouse/human chimeras.

    abstract::A human skin allograft injury model in immunodeficient mice, engrafted with human peripheral blood mononuclear cells from a different donor, has been used to test whether reagents that block human T cell CD2 interactions with its principal ligand, LFA-3 (CD58), can inhibit immune reactions in vivo. In this model, huma...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt0897-759

    authors: Sultan P,Schechner JS,McNiff JM,Hochman PS,Hughes CC,Lorber MI,Askenase PW,Pober JS

    更新日期:1997-08-01 00:00:00

  • Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences.

    abstract::Relatively low levels of expression from naturally occurring promoters have limited the use of muscle as a gene therapy target. Myogenic restricted gene promoters display complex organization usually involving combinations of several myogenic regulatory elements. By random assembly of E-box, MEF-2, TEF-1, and SRE site...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/6981

    authors: Li X,Eastman EM,Schwartz RJ,Draghia-Akli R

    更新日期:1999-03-01 00:00:00

  • Resistance gene cloning from a wild crop relative by sequence capture and association genetics.

    abstract::Disease resistance (R) genes from wild relatives could be used to engineer broad-spectrum resistance in domesticated crops. We combined association genetics with R gene enrichment sequencing (AgRenSeq) to exploit pan-genome variation in wild diploid wheat and rapidly clone four stem rust resistance genes. AgRenSeq ena...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/s41587-018-0007-9

    authors: Arora S,Steuernagel B,Gaurav K,Chandramohan S,Long Y,Matny O,Johnson R,Enk J,Periyannan S,Singh N,Asyraf Md Hatta M,Athiyannan N,Cheema J,Yu G,Kangara N,Ghosh S,Szabo LJ,Poland J,Bariana H,Jones JDG,Bentley AR,A

    更新日期:2019-02-01 00:00:00

  • Presymptomatic visualization of plant-virus interactions by thermography.

    abstract::Salicylic acid (SA), produced by plants as a signal in defense against pathogens, induces metabolic heating mediated by alternative respiration in flowers of thermogenic plants, and, when exogenously applied, increases leaf temperature in nonthermogenic plants. We have postulated that the latter phenomenon would be de...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/11765

    authors: Chaerle L,Van Caeneghem W,Messens E,Lambers H,Van Montagu M,Van Der Straeten D

    更新日期:1999-08-01 00:00:00

  • De novo assembly of soybean wild relatives for pan-genome analysis of diversity and agronomic traits.

    abstract::Wild relatives of crops are an important source of genetic diversity for agriculture, but their gene repertoire remains largely unexplored. We report the establishment and analysis of a pan-genome of Glycine soja, the wild relative of cultivated soybean Glycine max, by sequencing and de novo assembly of seven phylogen...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.2979

    authors: Li YH,Zhou G,Ma J,Jiang W,Jin LG,Zhang Z,Guo Y,Zhang J,Sui Y,Zheng L,Zhang SS,Zuo Q,Shi XH,Li YF,Zhang WK,Hu Y,Kong G,Hong HL,Tan B,Song J,Liu ZX,Wang Y,Ruan H,Yeung CK,Liu J,Wang H,Zhang LJ,Guan RX

    更新日期:2014-10-01 00:00:00

  • Epigenome-wide association data implicate DNA methylation as an intermediary of genetic risk in rheumatoid arthritis.

    abstract::Epigenetic mechanisms integrate genetic and environmental causes of disease, but comprehensive genome-wide analyses of epigenetic modifications have not yet demonstrated robust association with common diseases. Using Illumina HumanMethylation450 arrays on 354 anti-citrullinated protein antibody-associated rheumatoid a...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.2487

    authors: Liu Y,Aryee MJ,Padyukov L,Fallin MD,Hesselberg E,Runarsson A,Reinius L,Acevedo N,Taub M,Ronninger M,Shchetynsky K,Scheynius A,Kere J,Alfredsson L,Klareskog L,Ekström TJ,Feinberg AP

    更新日期:2013-02-01 00:00:00

  • MALDI-TOF based mutation detection using tagged in vitro synthesized peptides.

    abstract::Matrix-assisted laser desorption/ionization time-of-flight mass spectrometry (MALDI-TOF) is a powerful method to quickly and accurately determine the masses of peptides. Most genetic analyses, however, begin with PCR amplification of a test sequence to generate DNA, which is more difficult than peptides to analyze by ...

    journal_title:Nature biotechnology

    pub_type:

    doi:10.1038/72013

    authors: Garvin AM,Parker KC,Haff L

    更新日期:2000-01-01 00:00:00

  • Pancreatic endoderm derived from human embryonic stem cells generates glucose-responsive insulin-secreting cells in vivo.

    abstract::Development of a cell therapy for diabetes would be greatly aided by a renewable supply of human beta-cells. Here we show that pancreatic endoderm derived from human embryonic stem (hES) cells efficiently generates glucose-responsive endocrine cells after implantation into mice. Upon glucose stimulation of the implant...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt1393

    authors: Kroon E,Martinson LA,Kadoya K,Bang AG,Kelly OG,Eliazer S,Young H,Richardson M,Smart NG,Cunningham J,Agulnick AD,D'Amour KA,Carpenter MK,Baetge EE

    更新日期:2008-04-01 00:00:00

  • Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape.

    abstract::Delivery of short interfering RNAs (siRNAs) remains a key challenge in the development of RNA interference (RNAi) therapeutics. A better understanding of the mechanisms of siRNA cellular uptake, intracellular transport and endosomal release could critically contribute to the improvement of delivery methods. Here we mo...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.2612

    authors: Gilleron J,Querbes W,Zeigerer A,Borodovsky A,Marsico G,Schubert U,Manygoats K,Seifert S,Andree C,Stöter M,Epstein-Barash H,Zhang L,Koteliansky V,Fitzgerald K,Fava E,Bickle M,Kalaidzidis Y,Akinc A,Maier M,Zerial M

    更新日期:2013-07-01 00:00:00

  • Human feeders support prolonged undifferentiated growth of human inner cell masses and embryonic stem cells.

    abstract::Previous reports have demonstrated the growth of undifferentiated human embryonic stem (HES) cells on mouse embryonic fibroblast (MEF) feeders and on laminin- or Matrigel-coated plastic surfaces supplemented with MEF-conditioned medium. These xenosupport systems run the risk of cross-transfer of animal pathogens from ...

    journal_title:Nature biotechnology

    pub_type:

    doi:10.1038/nbt726

    authors: Richards M,Fong CY,Chan WK,Wong PC,Bongso A

    更新日期:2002-09-01 00:00:00

  • The complete genome sequence of the meat-borne lactic acid bacterium Lactobacillus sakei 23K.

    abstract::Lactobacillus sakei is a psychotrophic lactic acid bacterium found naturally on fresh meat and fish. This microorganism is widely used in the manufacture of fermented meats and has biotechnological potential in biopreservation and food safety. We have explored the 1,884,661-base-pair (bp) circular chromosome of strain...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt1160

    authors: Chaillou S,Champomier-Vergès MC,Cornet M,Crutz-Le Coq AM,Dudez AM,Martin V,Beaufils S,Darbon-Rongère E,Bossy R,Loux V,Zagorec M

    更新日期:2005-12-01 00:00:00

  • DNA variation and the future of human genetics.

    abstract::The use of DNA variants in the mapping of the human genome and in the positional cloning of monogenic disease genes is well established. Determining the genetic bases of the more common "multifactorial" diseases, however, presents a major challenge. The genetics of these diseases are complicated by the interplay betwe...

    journal_title:Nature biotechnology

    pub_type: 杂志文章,评审

    doi:10.1038/nbt0198-33

    authors: Schafer AJ,Hawkins JR

    更新日期:1998-01-01 00:00:00

  • Genetically engineered plants producing opines alter their biological environment.

    abstract::Little is known about the consequences of releasing genetically engineered plants (GEP) into the environment. Using opine-producing GEP, we show that transgenic plants alter their biological environment, more precisely the root-associated bacterial populations. The alterations were both transgene-specific and target p...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt0497-369

    authors: Oger P,Petit A,Dessaux Y

    更新日期:1997-04-01 00:00:00

  • Automated sorting of live transgenic embryos.

    abstract::The vast selection of Drosophila mutants is an extraordinary resource for exploring molecular events underlying development and disease. We have designed and constructed an instrument that automatically separates Drosophila embryos of one genotype from a larger population of embryos, based on a fluorescent protein mar...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/84422

    authors: Furlong EE,Profitt D,Scott MP

    更新日期:2001-02-01 00:00:00

  • An integrated mass spectrometric and computational framework for the analysis of protein interaction networks.

    abstract::Biological systems are controlled by protein complexes that associate into dynamic protein interaction networks. We describe a strategy that analyzes protein complexes through the integration of label-free, quantitative mass spectrometry and computational analysis. By evaluating peptide intensity profiles throughout t...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt1289

    authors: Rinner O,Mueller LN,Hubálek M,Müller M,Gstaiger M,Aebersold R

    更新日期:2007-03-01 00:00:00

  • Transgenic mice with defined combinations of drug-inducible reprogramming factors.

    abstract::Proviruses carrying drug-inducible Oct4, Sox2, Klf4 and c-Myc used to derive 'primary' induced pluripotent stem (iPS) cells were segregated through germline transmission, generating mice and cells carrying subsets of the reprogramming factors. Drug treatment produced 'secondary' iPS cells only when the missing factor ...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.1520

    authors: Markoulaki S,Hanna J,Beard C,Carey BW,Cheng AW,Lengner CJ,Dausman JA,Fu D,Gao Q,Wu S,Cassady JP,Jaenisch R

    更新日期:2009-02-01 00:00:00

  • Continuous evolution of SpCas9 variants compatible with non-G PAMs.

    abstract::The targeting scope of Streptococcus pyogenes Cas9 (SpCas9) and its engineered variants is largely restricted to protospacer-adjacent motif (PAM) sequences containing G bases. Here we report the evolution of three new SpCas9 variants that collectively recognize NRNH PAMs (where R is A or G and H is A, C or T) using ph...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/s41587-020-0412-8

    authors: Miller SM,Wang T,Randolph PB,Arbab M,Shen MW,Huang TP,Matuszek Z,Newby GA,Rees HA,Liu DR

    更新日期:2020-04-01 00:00:00

  • Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors.

    abstract::A major shortcoming to the use of adeno-associated virus (rAAV) vectors is their limited packaging size. To overcome this hurdle, we split an expression cassette and cloned it into two separate vectors. The vectors contained either a nuclear localizing Escherichia coli lacZ transgene (nlslacZ) with a splice acceptor, ...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/75390

    authors: Nakai H,Storm TA,Kay MA

    更新日期:2000-05-01 00:00:00

  • Controlled light-exposure microscopy reduces photobleaching and phototoxicity in fluorescence live-cell imaging.

    abstract::Fluorescence microscopy of living cells enables visualization of the dynamics and interactions of intracellular molecules. However, fluorescence live-cell imaging is limited by photobleaching and phototoxicity induced by the excitation light. Here we describe controlled light-exposure microscopy (CLEM), a simple imagi...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt1278

    authors: Hoebe RA,Van Oven CH,Gadella TW Jr,Dhonukshe PB,Van Noorden CJ,Manders EM

    更新日期:2007-02-01 00:00:00

  • Erect leaves caused by brassinosteroid deficiency increase biomass production and grain yield in rice.

    abstract::New cultivars with very erect leaves, which increase light capture for photosynthesis and nitrogen storage for grain filling, may have increased grain yields. Here we show that the erect leaf phenotype of a rice brassinosteroid-deficient mutant, osdwarf4-1, is associated with enhanced grain yields under conditions of ...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt1173

    authors: Sakamoto T,Morinaka Y,Ohnishi T,Sunohara H,Fujioka S,Ueguchi-Tanaka M,Mizutani M,Sakata K,Takatsuto S,Yoshida S,Tanaka H,Kitano H,Matsuoka M

    更新日期:2006-01-01 00:00:00

  • Metabolic redesign of vitamin E biosynthesis in plants for tocotrienol production and increased antioxidant content.

    abstract::Tocotrienols are the primary form of vitamin E in seeds of most monocot plants, including cereals such as rice and wheat. As potent antioxidants, tocotrienols contribute to the nutritive value of cereal grains in human and livestock diets. cDNAs encoding homogentisic acid geranylgeranyl transferase (HGGT), which catal...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt853

    authors: Cahoon EB,Hall SE,Ripp KG,Ganzke TS,Hitz WD,Coughlan SJ

    更新日期:2003-09-01 00:00:00

  • NRT1.1B is associated with root microbiota composition and nitrogen use in field-grown rice.

    abstract::Nitrogen-use efficiency of indica varieties of rice is superior to that of japonica varieties. We apply 16S ribosomal RNA gene profiling to characterize root microbiota of 68 indica and 27 japonica varieties grown in the field. We find that indica and japonica recruit distinct root microbiota. Notably, indica-enriched...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/s41587-019-0104-4

    authors: Zhang J,Liu YX,Zhang N,Hu B,Jin T,Xu H,Qin Y,Yan P,Zhang X,Guo X,Hui J,Cao S,Wang X,Wang C,Wang H,Qu B,Fan G,Yuan L,Garrido-Oter R,Chu C,Bai Y

    更新日期:2019-06-01 00:00:00

  • MicroRNA-mediated species-specific attenuation of influenza A virus.

    abstract::Influenza A virus leads to yearly epidemics and sporadic pandemics. Present prophylactic strategies focus on egg-grown, live, attenuated influenza vaccines (LAIVs), in which attenuation is generated by conferring temperature sensitivity onto the virus. Here we describe an alternative approach to attenuating influenza ...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.1542

    authors: Perez JT,Pham AM,Lorini MH,Chua MA,Steel J,tenOever BR

    更新日期:2009-06-01 00:00:00

  • Transient inhibition of BMP signaling by Noggin induces cardiomyocyte differentiation of mouse embryonic stem cells.

    abstract::Embryonic stem (ES) cells are a promising source of cardiomyocytes, but clinical application of ES cells has been hindered by the lack of reliable selective differentiation methods. Differentiation into any lineage is partly dependent on the regulatory mechanisms of normal early development. Although several signals, ...

    journal_title:Nature biotechnology

    pub_type: 信件

    doi:10.1038/nbt1093

    authors: Yuasa S,Itabashi Y,Koshimizu U,Tanaka T,Sugimura K,Kinoshita M,Hattori F,Fukami S,Shimazaki T,Ogawa S,Okano H,Fukuda K

    更新日期:2005-05-01 00:00:00

  • Lipid production in Nannochloropsis gaditana is doubled by decreasing expression of a single transcriptional regulator.

    abstract::Lipid production in the industrial microalga Nannochloropsis gaditana exceeds that of model algal species and can be maximized by nutrient starvation in batch culture. However, starvation halts growth, thereby decreasing productivity. Efforts to engineer N. gaditana strains that can accumulate biomass and overproduce ...

    journal_title:Nature biotechnology

    pub_type: 杂志文章

    doi:10.1038/nbt.3865

    authors: Ajjawi I,Verruto J,Aqui M,Soriaga LB,Coppersmith J,Kwok K,Peach L,Orchard E,Kalb R,Xu W,Carlson TJ,Francis K,Konigsfeld K,Bartalis J,Schultz A,Lambert W,Schwartz AS,Brown R,Moellering ER

    更新日期:2017-07-01 00:00:00