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Ryszard Kole

Research Profile

Chemicals & Drugs

Physiology

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Sue Fletcher; Clayton Fragall; Kane Greer; Russell Johnsen; Ryszard Kole; Penny Meloni; Francesco Muntoni; Carl F Adkin; Brenda Wong; Steve D Wilton
Targeted exon skipping to address "leaky" mutations in the dystrophin gene.
Sebahattin Cirak; George Dickson; Lucy Feng; Maria Elena Garralda; Michela Guglieri; Ryszard Kole; Jennifer E Morgan; Stephen Abbs; Karen Anthony; Virginia Arechavala-Gomeza; John Bourke; Kate Bushby; Caroline Sewry; Stephen B Shrewsbury; Volker Straub; Silvia Torelli; Dominic J Wells; Stephen D Wilton; Matthew J A Wood; Francesco Muntoni
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
John A Bauman; Ryszard Kole
Modulation of RNA splicing as a potential treatment for cancer.

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2012: Sue Fletcher; Clayton Fragall; Kane Greer; Russell Johnsen; Ryszard Kole; Penny Meloni; Francesco Muntoni; Carl F Adkin; Brenda Wong; Steve D Wilton
Targeted exon skipping to address "leaky" mutations in the dystrophin gene.
Molecular therapy. Nucleic acids 2012;1():e48.
2011: Sebahattin Cirak; George Dickson; Lucy Feng; Maria Elena Garralda; Michela Guglieri; Ryszard Kole; Jennifer E Morgan; Stephen Abbs; Karen Anthony; Virginia Arechavala-Gomeza; John Bourke; Kate Bushby; Caroline Sewry; Stephen B Shrewsbury; Volker Straub; Silvia Torelli; Dominic J Wells; Stephen D Wilton; Matthew J A Wood; Francesco Muntoni
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Lancet 2011;378(9791):595-605.
2011: John A Bauman; Ryszard Kole
Modulation of RNA splicing as a potential treatment for cancer.
Bioengineered bugs 2011;2(3):125-8.
2011: Clayton T Fragall; Russell D Johnsen; Ryszard Kole; Abbie M Adams; Sue Fletcher; Steve D Wilton
Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching.
BMC medical genetics 2011;12():141.
2010: John A Bauman; Shyh-Dar Li; Angela Yang; Leaf Huang; Ryszard Kole
Anti-tumor activity of splice-switching oligonucleotides.
Nucleic acids research 2010;38(22):8348-56.
2010: Natee Jearawiriyapaisarn; Hong M Moulton; Peter Sazani; Ryszard Kole; Monte Willis
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.
Cardiovascular research 2010;85(3):444-53.
2010: Aurélie Goyenvalle; Arran Babbs; Dave Powell; Ryszard Kole; Sue Fletcher; Steve D Wilton; Kay E Davies
Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
Molecular therapy : the journal of the American Society of Gene Therapy 2010;18(1):198-205.
2009: Maria Kinali; Virginia Arechavala-Gomeza; Lucy Feng; Sebahattin Cirak; David Hunt; Carl Adkin; Michela Guglieri; Emma Ashton; Stephen Abbs; Petros Nihoyannopoulos; Maria Elena Garralda; Mary Rutherford; Caroline McCulley; Linda Popplewell; Ian R Graham; George Dickson; Matthew J A Wood; Dominic J Wells; Steve D Wilton; Ryszard Kole; Volker Straub; Kate Bushby; Caroline Sewry; Jennifer E Morgan; Francesco Muntoni
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.
Lancet neurology 2009;8(10):918-28.
2009: Hong M Moulton; Bo Wu; Natee Jearawiriyapaisarn; Peter Sazani; Qi Long Lu; Ryszard Kole
Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy.
Annals of the New York Academy of Sciences 2009;1175():55-60.
2009: Pallavi S Lonkar; Ki-Hyun Kim; Jean Y Kuan; Joanna Y Chin; Faye A Rogers; Melissa P Knauert; Ryszard Kole; Peter E Nielsen; Peter M Glazer
Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.
Nucleic acids research 2009;37(11):3635-44.
2009: Lamar Mair; Kris Ford; Mohammed Alam; Ryszard Kole; Michael Fisher; Joy van Baren
Size-uniform 200 nm particles: fabrication and application to magnetofection.
Journal of biomedical nanotechnology 2009;5(2):182-91.
2009: John Bauman; Natee Jearawiriyapaisarn; Ryszard Kole
Therapeutic potential of splice-switching oligonucleotides.
Oligonucleotides 2009;19(1):1-13.
2009: Jing Wan; Peter Sazani; Ryszard Kole
Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells.
International journal of cancer. Journal international du cancer 2009;124(4):772-7.
2009: Saovaros Svasti; Thipparat Suwanmanee; Suthat Fucharoen; Hong M Moulton; Michelle H Nelson; Nobuyo Maeda; Oliver Smithies; Ryszard Kole
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.
Proceedings of the National Academy of Sciences of the United States of America 2009;106(4):1205-10.
2008: Joanna Y Chin; Jean Y Kuan; Pallavi S Lonkar; Diane S Krause; Michael M Seidman; Kenneth R Peterson; Peter E Nielsen; Ryszard Kole; Peter M Glazer
Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids.
Proceedings of the National Academy of Sciences of the United States of America 2008;105(36):13514-9.
2008: Natee Jearawiriyapaisarn; Hong M Moulton; Brian Buckley; Jennifer Roberts; Peter Sazani; Suthat Fucharoen; Patrick L Iversen; Ryszard Kole
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(9):1624-9.
2008: Maria A Graziewicz; Teresa Tarrant; Brian Buckley; Jennifer Roberts; LeShara Fulton; Henrik Hansen; Henrik Ørum; Ryszard Kole; Peter Sazani
An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(7):1316-22.
2007: Nanfei Zhang; James J Peairs; Ping Yang; Jillian Tyrrell; Jennifer Roberts; Ryszard Kole; Glenn J Jaffe
The importance of Bcl-xL in the survival of human RPE cells.
Investigative ophthalmology & visual science 2007;48(8):3846-53.
2007: Sarah Resina; Ryszard Kole; Adrian Travo; Bernard Lebleu; Alain THIERRY
Switching on transgene expression by correcting aberrant splicing using multi-targeting steric-blocking oligonucleotides.
The journal of gene medicine 2007;9(6):498-510.
2006: Jennifer Roberts; Enzo Palma; Peter Sazani; Henrik Ørum; Moo Je Cho; Ryszard Kole
Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2006;14(4):471-5.
2006: Tiffany Williams; Ryszard Kole
Analysis of prostate-specific membrane antigen splice variants in LNCap cells.
Oligonucleotides 2006;16(2):186-95.
2004: Ryszard Kole; Tiffany Williams; Lisa Cohen
RNA modulation, repair and remodeling by splice switching oligonucleotides.
Acta biochimica Polonica 2004;51(2):373-8.
2004: Ryszard Kole; Marla Vacek; Tiffany Williams
Modification of alternative splicing by antisense therapeutics.
Oligonucleotides 2004;14(1):65-74.
2003: Cynthia L Bristow; Danielle Carrick; Ryszard Kole
HIV-1 preferentially binds receptors copatched with cell-surface elastase.
Blood 2003;102(13):4479-86.
2003: Andrew J Ghio; Eva Nozik-Grayck; Jennifer L Turi; Ilona Jaspers; Danielle Carrick; Ryszard Kole; Claude A Piantadosi
Superoxide-dependent iron uptake: a new role for anion exchange protein 2.
American journal of respiratory cell and molecular biology 2003;29(6):653-60.
2003: Peter Sazani; Ryszard Kole
Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.
The Journal of clinical investigation 2003;112(4):481-6.
2003: Marla M Vacek; Peter Sazani; Ryszard Kole
Antisense-mediated redirection of mRNA splicing.
Cellular and molecular life sciences : CMLS 2003;60(5):825-33.
2003: Peter Sazani; Anna Astriab-Fischer; Ryszard Kole
Effects of base modifications on antisense properties of 2'-O-methoxyethyl and PNA oligonucleotides.
Antisense & nucleic acid drug development 2003;13(3):119-28.
2003: Peter Sazani; Ryszard Kole
Modulation of alternative splicing by antisense oligonucleotides.
Progress in molecular and subcellular biology 2003;31():217-39.
2003: Marla M Vacek; Hong Ma; Federica Gemignani; Giuseppina Lacerra; Tal Kafri; Ryszard Kole
High-level expression of hemoglobin A in human thalassemic erythroid progenitor cells following lentiviral vector delivery of an antisense snRNA.
Blood 2003;101(1):104-11.
2002: Danielle Carrick; James L Mohler; Ryszard Kole
Cellular response to an antisense-mediated shift of Bcl-x pre-mRNA splicing and antineoplastic agents.
The Journal of biological chemistry 2002;277(51):49374-82.
2002: Thipparat Suwanmanee; Halina Sierakowska; Suthat Fucharoen; Ryszard Kole
Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder.
Molecular therapy : the journal of the American Society of Gene Therapy 2002;6(6):718-26.
2002: Peter Sazani; Federica Gemignani; Shin-Hong Kang; Martin A Maier; Muthiah Manoharan; Magnus Persmark; Donna Bortner; Ryszard Kole
Systemically delivered antisense oligomers upregulate gene expression in mouse tissues.
Nature biotechnology 2002;20(12):1228-33.
2002: Federica Gemignani; Peter Sazani; Paul Morcos; Ryszard Kole
Temperature-dependent splicing of beta-globin pre-mRNA.
Nucleic acids research 2002;30(21):4592-8.
2002: Peter Sazani; Marla M Vacek; Ryszard Kole
Short-term and long-term modulation of gene expression by antisense therapeutics.
Current opinion in biotechnology 2002;13(5):468-72.
2002: Thipparat Suwanmanee; Halina Sierakowska; Giuseppina Lacerra; Saovaros Svasti; Suzanne L Kirby; Christopher E Walsh; Suthat Fucharoen; Ryszard Kole
Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.
Molecular pharmacology 2002;62(3):545-53.
2002: Danielle Carrick; Ryszard Kole
Control of alternative splicing by antisense oligonucleotides as a potential chemotherapy: effects on gene expression.
Biochimica et biophysica acta 2002;1587(2-3):126-32.
2001: Federica Gemignani; S Landi; D M DeMarini; Ryszard Kole
Spontaneous and MNNG-induced reversion of an EGFP construct in HeLa cells: an assay for observing mutations in living cells by fluorescent microscopy.
Human mutation 2001;18(6):526-34.
2001: Danielle Carrick; Peter Sazani; Ryszard Kole
Modification of alternative splicing by antisense oligonucleotides as a potential chemotherapy for cancer and other diseases.
Current cancer drug targets 2001;1(3):211-30.
2001: Peter Sazani; S H Kang; M A Maier; C Wei; J Dillman; J Summerton; Muthiah Manoharan; Ryszard Kole
Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs.
Nucleic acids research 2001;29(19):3965-74.
2001: H Tian; Ryszard Kole
Strong RNA splicing enhancers identified by a modified method of cycled selection interact with SR protein.
The Journal of biological chemistry 2001;276(36):33833-9.
2001: Ryszard Kole; Peter Sazani
Antisense effects in the cell nucleus: modification of splicing.
Current opinion in molecular therapeutics 2001;3(3):229-34.
2001: Danielle Carrick; Carl Bortner; J A Cidlowski; Ryszard Kole
Modification of alternative splicing of Bcl-x pre-mRNA in prostate and breast cancer cells. analysis of apoptosis and cell death.
The Journal of biological chemistry 2001;276(19):16411-7.
2000: L Gorman; Danielle Carrick; Ryszard Kole
Restoration of correct splicing of thalassemic beta-globin pre-mRNA by modified U1 snRNAs.
The Journal of biological chemistry 2000;275(46):35914-9.
2000: Giuseppina Lacerra; Halina Sierakowska; Clementina Carestia; Suthat Fucharoen; J Summerton; D Weller; Ryszard Kole
Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.
Proceedings of the National Academy of Sciences of the United States of America 2000;97(17):9591-6.
2000: Danielle Carrick; Ryszard Kole
Modification of alternative splicing pathways as a potential approach to chemotherapy.
Pharmacology & therapeutics 2000;85(3):237-43.
2000: Halina Sierakowska; L Gorman; S H Kang; Ryszard Kole
Antisense oligonucleotides and RNAs as modulators of pre-mRNA splicing.
Methods in enzymology 2000;313():506-21.
2000: Halina Sierakowska; S Agrawal; Ryszard Kole
Antisense oligonucleotides as modulators of pre-mRNA splicing.
Methods in molecular biology (Clifton, N.J.) 2000;133():223-33.
1999: Kenneth Friedman; Jolanta Kole; Jonathan A Cohn; Michael Knowles; Lawrence M Silverman; Ryszard Kole
Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides.
The Journal of biological chemistry 1999;274(51):36193-9.
1999: S H Kang; E L Zirbes; Ryszard Kole
Delivery of antisense oligonucleotides and plasmid DNA with various carrier agents.
Antisense & nucleic acid drug development 1999;9(6):497-505.
1999: D Suter; R Tomasini; U Reber; L Gorman; Ryszard Kole; Daniel Schümperli
Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human beta-thalassemic mutations.
Human molecular genetics 1999;8(13):2415-23.
1999: Robert DeLong; Hoon Yoo; Suresh K Alahari; M Fisher; SM Short; S H Kang; Ryszard Kole; V Janout; S L Regan; Rudy L Juliano
Novel cationic amphiphiles as delivery agents for antisense oligonucleotides.
Nucleic acids research 1999;27(16):3334-41.
1999: G Schmajuk; Halina Sierakowska; Ryszard Kole
Antisense oligonucleotides with different backbones. Modification of splicing pathways and efficacy of uptake.
The Journal of biological chemistry 1999;274(31):21783-9.
1999: Stephen D Wilton; F Lloyd; Kylie S Carville; Susan Fletcher; Kaite Honeyman; S Agrawal; Ryszard Kole
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.
Neuromuscular disorders : NMD 1999;9(5):330-8.
1999: Rudy L Juliano; Suresh K Alahari; Hoon Yoo; Ryszard Kole; Moo Je Cho
Antisense pharmacodynamics: critical issues in the transport and delivery of antisense oligonucleotides.
Pharmaceutical research 1999;16(4):494-502.
1999: Halina Sierakowska; M J Sambade; Daniel Schümperli; Ryszard Kole
Sensitivity of splice sites to antisense oligonucleotides in vivo.
RNA (New York, N.Y.) 1999;5(3):369-77.
1998: Eva Westphal; Halina Sierakowska; E Livanos; Ryszard Kole; Jean-Michel H Vos
A system for shuttling 200-kb BAC/PAC clones into human cells: stable extrachromosomal persistence and long-term ectopic gene activation.
Human gene therapy 1998;9(13):1863-73.
1998: S H Kang; Moo Je Cho; Ryszard Kole
Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development.
Biochemistry 1998;37(18):6235-9.
1998: L Gorman; D Suter; V Emerick; Daniel Schümperli; Ryszard Kole
Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs.
Proceedings of the National Academy of Sciences of the United States of America 1998;95(9):4929-34.
1998: Jada Lewis; B Yang; R Kim; Halina Sierakowska; Ryszard Kole; Oliver Smithies; Nobuyo Maeda
A common human beta globin splicing mutation modeled in mice.
Blood 1998;91(6):2152-6.
1997: Ryszard Kole
Modification of pre-mRNA splicing by antisense oligonucleotides.
Acta biochimica Polonica 1997;44(2):231-7.
1996: Halina Sierakowska; M J Sambade; S Agrawal; Ryszard Kole
Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides.
Proceedings of the National Academy of Sciences of the United States of America 1996;93(23):12840-4.
1996: Zbigniew Dominski; Ryszard Kole
Effects of exon sequences on splicing of model pre-mRNA substrates in vitro.
Acta biochimica Polonica 1996;43(1):161-73.
1996: Zbigniew Dominski; P Ferree; Ryszard Kole
Antisense 2'-O-methyloligoribonucleotides hybridized to RNA block a nuclear, ATP-dependent 3'-5' exonuclease.
Antisense & nucleic acid drug development 1996;6(1):37-45.
1995: H Tian; Ryszard Kole
Selection of novel exon recognition elements from a pool of random sequences.
Molecular and cellular biology 1995;15(11):6291-8.
1995: H X Liu; G J Goodall; Ryszard Kole; Witold Filipowicz
Effects of secondary structure on pre-mRNA splicing: hairpins sequestering the 5' but not the 3' splice site inhibit intron processing in Nicotiana plumbaginifolia.
The EMBO journal 1995;14(2):377-88.
1994: Zbigniew Dominski; Ryszard Kole
Identification and characterization by antisense oligonucleotides of exon and intron sequences required for splicing.
Molecular and cellular biology 1994;14(11):7445-54.
1994: Zbigniew Dominski; Ryszard Kole
Identification of exon sequences involved in splice site selection.
The Journal of biological chemistry 1994;269(38):23590-6.
1994: Maria F Czyzyk-Krzeska; Zbigniew Dominski; Ryszard Kole; David E Millhorn
Hypoxia stimulates binding of a cytoplasmic protein to a pyrimidine-rich sequence in the 3'-untranslated region of rat tyrosine hydroxylase mRNA.
The Journal of biological chemistry 1994;269(13):9940-5.
1993: Zbigniew Dominski; Ryszard Kole
Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.
Proceedings of the National Academy of Sciences of the United States of America 1993;90(18):8673-7.
1992: Zbigniew Dominski; Ryszard Kole
Cooperation of pre-mRNA sequence elements in splice site selection.
Molecular and cellular biology 1992;12(5):2108-14.
1992: U Utans; S E Behrens; R Lührmann; Ryszard Kole; Angela Krämer
A splicing factor that is inactivated during in vivo heat shock is functionally equivalent to the [U4/U6.U5] triple snRNP-specific proteins.
Genes & development 1992;6(4):631-41.
1991: Zbigniew Dominski; Ryszard Kole
Selection of splice sites in pre-mRNAs with short internal exons.
Molecular and cellular biology 1991;11(12):6075-83.
1991: S Akhtar; Ryszard Kole; Rudy L Juliano
Stability of antisense DNA oligodeoxynucleotide analogs in cellular extracts and sera.
Life sciences 1991;49(24):1793-801.
1990: R R Shukla; Zbigniew Dominski; T Zwierzynski; Ryszard Kole
Inactivation of splicing factors in HeLa cells subjected to heat shock.
The Journal of biological chemistry 1990;265(33):20377-83.
1989: P J Furdon; Zbigniew Dominski; Ryszard Kole
RNase H cleavage of RNA hybridized to oligonucleotides containing methylphosphonate, phosphorothioate and phosphodiester bonds.
Nucleic acids research 1989;17(22):9193-204.
1989: Halina Sierakowska; R R Shukla; Zbigniew Dominski; Ryszard Kole
Inhibition of pre-mRNA splicing by 5-fluoro-, 5-chloro-, and 5-bromouridine.
The Journal of biological chemistry 1989;264(32):19185-91.
1988: P J Furdon; Ryszard Kole
The length of the downstream exon and the substitution of specific sequences affect pre-mRNA splicing in vitro.
Molecular and cellular biology 1988;8(2):860-6.
1987: P J Furdon; Ryszard Kole
RNA fingerprinting using a small horizontal agarose gel electrophoresis apparatus.
Analytical biochemistry 1987;162(1):74-9.
1986: Halina Sierakowska; W Szer; P J Furdon; Ryszard Kole
Antibodies to hnRNP core proteins inhibit in vitro splicing of human beta-globin pre-mRNA.
Nucleic acids research 1986;14(13):5241-54.
1986: P J Furdon; Ryszard Kole
Inhibition of splicing but not cleavage at the 5' splice site by truncating human beta-globin pre-mRNA.
Proceedings of the National Academy of Sciences of the United States of America 1986;83(4):927-31.
1982: Ryszard Kole; S M Weissman
Accurate in vitro splicing of human beta-globin RNA.
Nucleic acids research 1982;10(18):5429-45.
1981: Ryszard Kole; Sidney Altman
Properties of purified ribonuclease P from Escherichia coli.
Biochemistry 1981;20(7):1902-6.
1980: Ryszard Kole; M F Baer; B C Stark; Sidney Altman
E. coli RNAase P has a required RNA component.
Cell 1980;19(4):881-7.
1979: Ryszard Kole; Sidney Altman
Reconstitution of RNase P activity from inactive RNA and protein.
Proceedings of the National Academy of Sciences of the United States of America 1979;76(8):3795-9.
1978: B C Stark; Ryszard Kole; E J Bowman; Sidney Altman
Ribonuclease P: an enzyme with an essential RNA component.
Proceedings of the National Academy of Sciences of the United States of America 1978;75(8):3717-21.
1976: Ryszard Kole; H Sierakowska; D Shugar
Novel activity of potato nucleotide pyrophosphatase.
Biochimica et biophysica acta 1976;438(2):540-50.
1974: Ryszard Kole; H Sierakowska; H Szemplińska; D Shugar
Mung bean nuclease: mode of action and specificity vs synthetic esters of 3'-nucleotides.
Nucleic acids research 1974;1(5):699-706.
1972: Ryszard Kole; H Sierakowska; D Shugar
A specific colorimetric and cytochemical substrate for ribonuclease T 2 : adenosine-3'-( -naphtylphosphate).
Biochimica et biophysica acta 1972;289(2):323-30.
1971: Ryszard Kole; H Sierakowska; D Shugar
A ribonuclease substrate resistant to phosphodiesterase II: 5'-O-benzyluridine-3'-(alpha-naphthylphosphate).
Biochemical and biophysical research communications 1971;44(6):1482-7.
1971: Ryszard Kole; H Sierakowska
-naphthyl ribonucleoside-3' phosphate analogues for the cytochemical and colorimetric assay of ribonucleases.
Acta biochimica Polonica 1971;18(2):187-97.