Gene therapy and intracytoplasmatic sperm injection (ICSI) - a review.

Human gene therapy (HGT), the treatment or prevention of disease by gene transfer is, regarded by many, as a potential revolution in medicine, because gene therapies target the causes of disease, whereas most current drugs treat the symptoms. Micro-assisted fertilization in the form of intracytoplasmatic sperm injection (ICSI) has truly revolutionized the treatment options for couples with impaired semen quality, and those with both obstructive and non-obstructive azoospermia. ICSI involves the injection of a single sperm directly into the cytoplasm of a mature egg (oocyte) using a glass needle (pipette). Fertilization with this technique occurs in 50%-80% of injected oocytes, but may damage a small percentage of them. With gene therapy, there are new and varied strategies for gene transfer and genome sequence manipulation with improved methodologies that use the technique of microinjection such as the intracytoplasmatic sperm injection-mediated transgenesis (ICSI-Tr), active transgenesis or the pronuclear microinjection technique. This review will look at these methods as well as their potential applications and limitations.

[1]  P. Mombaerts,et al.  Efficient metaphase II transgenesis with different transgene archetypes , 2001, Nature Biotechnology.

[2]  A. Beaudet,et al.  Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity , 1998, Nature Genetics.

[3]  S. Moisyadi,et al.  Recombinase-mediated mouse transgenesis by intracytoplasmic sperm injection. , 2005, Theriogenology.

[4]  R. Samulski,et al.  Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'γ)2 antibody , 1999, Nature Biotechnology.

[5]  D. Cumberland,et al.  Ultrasound enhances reporter gene expression after transfection of vascular cells in vitro. , 1999, Circulation.

[6]  S. Moisyadi,et al.  Tn5 Transposase-Mediated Mouse Transgenesis1 , 2005, Biology of reproduction.

[7]  A. van der Eb,et al.  New helper cells and matched early region 1-deleted adenovirus vectors prevent generation of replication-competent adenoviruses. , 1998, Human gene therapy.

[8]  N. Naumann,et al.  The efficacy of local anaesthesia for percutaneous epididymal sperm aspiration and testicular sperm aspiration. , 1998, Human reproduction.

[9]  T. Bonetti,et al.  Fertilization and pregnancy outcome after intracytoplasmic injection with fresh or cryopreserved ejaculated spermatozoa. , 2007, Fertility and sterility.

[10]  M. Hattori,et al.  Production of inbred and hybrid transgenic mice carrying large (> 200 kb) foreign DNA fragments by intracytoplasmic sperm injection , 2005, Molecular reproduction and development.

[11]  D. Huhn,et al.  Efficient gene transfer into lymphoma cells using adenoviral vectors combined with lipofection , 2000, Cancer Gene Therapy.

[12]  Y. Soong,et al.  A successful pregnancy after subzonal insertion with epididymal sperm and coculture on Vero cell monolayer. , 1993, Fertility and sterility.

[13]  J. Wolff,et al.  The Cambrian period of nonviral gene delivery , 1998, Nature Biotechnology.

[14]  B. Carter.  Gene therapy as drug development. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

[15]  K. Lundin,et al.  Sperm retrieval and fertilization in repeated percutaneous epididymal sperm aspiration. , 1998, Human reproduction.

[16]  W. Anderson,et al.  Human gene therapy. , 1992, Science.

[17]  A. Gutiérrez-Adán,et al.  Efficient Generation of Transgenic Mice with Intact Yeast Artificial Chromosomes by Intracytoplasmic Sperm Injection1 , 2004, Biology of reproduction.

[18]  G. Acsadi,et al.  Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. , 1992, Human molecular genetics.

[19]  K. Propert,et al.  Immune responses to adenovirus and adeno-associated virus in humans , 1999, Gene Therapy.

[20]  G. Nabel,et al.  Immunotherapy of malignancy by in vivo gene transfer into tumors. , 1993, Proceedings of the National Academy of Sciences of the United States of America.

[21]  J. Tesarik,et al.  Treatment of severe male infertility by micromanipulation-assisted fertilization: an update. , 2007, Frontiers in bioscience : a journal and virtual library.

[22]  D. Bohl,et al.  Selective uptake and sustained expression of AAV vectors following subcutaneous delivery , 1999, The journal of gene medicine.

[23]  R. Johnson,et al.  Humoral and cellular immune responses of nonhuman primates to long-term repeated lung exposure to Ad2/CFTR-2. , 1996, Gene therapy.

[24]  P. Felgner,et al.  Cationic liposome-mediated transfection , 1989, Nature.

[25]  S. Kuriyama,et al.  Expression of a retrovirally transduced gene under control of an internal housekeeping gene promoter does not persist due to methylation and is restored partially by 5-azacytidine treatment , 1998, Gene Therapy.

[26]  T. Flotte,et al.  Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus , 1998, The Lancet.

[27]  L. Smith,et al.  A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells. , 1996, Gene therapy.

[28]  S. Fishel,et al.  Subzonal insemination for the alleviation of infertility. , 1990, Fertility and sterility.

[29]  M. Haine,et al.  Van Damme A. , 1986 .

[30]  N. Yang,et al.  Gene gun and other non-viral approaches for cancer gene therapy , 1995, Nature Medicine.

[31]  M. Bureau,et al.  High-efficiency gene transfer into skeletal muscle mediated by electric pulses. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[32]  R Yanagimachi,et al.  Microsurgical injection of spermatozoa into hamster eggs with subsequent transformation of sperm nuclei into male pronuclei. , 1976, Biology of reproduction.

[33]  J. Isner,et al.  Direct intramuscular gene transfer of naked DNA encoding vascular endothelial growth factor augments collateral development and tissue perfusion. , 1996, Circulation.

[34]  P. Robbins,et al.  Viral vectors for gene therapy. , 1998, Trends in biotechnology.

[35]  J. Mulhall,et al.  Fertilization and pregnancy using intentionally cryopreserved testicular tissue as the sperm source for intracytoplasmic sperm injection in 10 men with non-obstructive azoospermia. , 1997, Human reproduction.

[36]  リュウゾウ ヤナギマチ.  Mammalian transgenesis by intracytoplasmic sperm injection , 1999 .

[37]  M. Cotten,et al.  Transferrin-polycation conjugates as carriers for DNA uptake into cells. , 1990, Proceedings of the National Academy of Sciences of the United States of America.

[38]  Simon C Watkins,et al.  Microbubbles targeted to intercellular adhesion molecule-1 bind to activated coronary artery endothelial cells. , 1998, Circulation.

[39]  J. Isner,et al.  Direct intramuscular injection of plasmid DNA encoding angiopoietin-1 but not angiopoietin-2 augments revascularization in the rabbit ischemic hindlimb. , 1998, Circulation.

[40]  Megumi Kato,et al.  Direct comparison between ICSI-mediated DNA transfer and pronuclear DNA microinjection for producing transgenic rats. , 2008, Experimental animals.

[41]  N. Zaninovic,et al.  Testicular sperm extraction with intracytoplasmic sperm injection for nonobstructive azoospermia. , 1997, Urology.

[42]  A. Gutiérrez-Adán,et al.  Effect of Transgene Concentration, Flanking Matrix Attachment Regions, and RecA-Coating on the Efficiency of Mouse Transgenesis Mediated by Intracytoplasmic Sperm Injection1 , 2007, Biology of reproduction.

[43]  Z. Nagy,et al.  High fertilization and pregnancy rate after intracytoplasmic sperm injection with spermatozoa obtained from testicle biopsy. , 1995, Human reproduction.

[44]  D. Gill,et al.  Towards gene therapy for cystic fibrosis: a clinical progress report , 1998, Gene Therapy.

[45]  J. Whelan,et al.  Progress in transcriptionally targeted and regulatable vectors for genetic therapy. , 1997, Human gene therapy.

[46]  B. Carter.  Viral Vectors for Gene Therapy , 2001 .

[47]  G. Serour,et al.  Intracytoplasmic sperm injection using microsurgically retrieved epididymal and testicular sperm. , 1996, Fertility and sterility.

[48]  H. Tournaye,et al.  Testicular sperm recovery in nine 47,XXY Klinefelter patients. , 1996, Human reproduction.

[49]  L. Huang,et al.  In vivo gene transfer via intravenous administration of cationic lipid–protamine–DNA (LPD) complexes , 1997, Gene Therapy.

[50]  E. Wolf,et al.  Efficient transgenesis in farm animals by lentiviral vectors , 2003, EMBO reports.

[51]  H E Malter,et al.  Partial zona dissection of the human oocyte: a nontraumatic method using micromanipulation to assist zona pellucida penetration. , 1989, Fertility and sterility.

[52]  Simon C Watkins,et al.  DNA–based immunization by in vivo transfection of dendritic cells , 1996, Nature Medicine.

[53]  C. Balagué,et al.  Adeno-associated virus Rep78 protein and terminal repeats enhance integration of DNA sequences into the cellular genome , 1997, Journal of virology.

[54]  H. Tournaye.  Surgical sperm recovery for intracytoplasmic sperm injection: which method is to be preferred? , 1999, Human reproduction.

[55]  G. Lukács,et al.  Analysis of differential lipofection efficiency in primary and established myoblasts. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[56]  D Mitchell,et al.  Partial zona dissection of human oocytes when failure of zona pellucida penetration is anticipated. , 1989, Human reproduction.

[57]  J. Fulka,et al.  Mouse oocyte maturation: the effect of modified nucleocytoplasmic ratio. , 1998, Reproduction, nutrition, development.

[58]  R. Crystal,et al.  Biologic bypass with the use of adenovirus-mediated gene transfer of the complementary deoxyribonucleic acid for vascular endothelial growth factor 121 improves myocardial perfusion and function in the ischemic porcine heart. , 1998, The Journal of thoracic and cardiovascular surgery.

[59]  D Needham,et al.  Increased microvascular permeability contributes to preferential accumulation of Stealth liposomes in tumor tissue. , 1993, Cancer research.

[60]  A. C. Perry,et al.  Hijacking oocyte DNA repair machinery in transgenesis? , 2000, Molecular reproduction and development.

[61]  Y. Kojima,et al.  Gene transfer to sperm and testis: future prospects of gene therapy for male infertility. , 2008, Current gene therapy.

[62]  M. Kurome,et al.  Effects of sperm pretreatment on efficiency of ICSI-mediated gene transfer in pigs. , 2007, The Journal of reproduction and development.

[63]  S. Payne,et al.  Open versus closed epididymal sperm retrieval in men with secondarily obstructed vasal systems--a preliminary report. , 1996, British journal of urology.

[64]  D. Geddes,et al.  Prospects for gene therapy in cystic fibrosis , 1999, Molecular medicine today.

[65]  J W Gordon,et al.  Assisted fertilization by zona drilling: a mouse model for correction of oligospermia. , 1986, The Journal of experimental zoology.

[66]  S. Antinori,et al.  Presentation of six pregnancies established by sub-zonal insemination (SUZI). , 1991, Human reproduction.

[67]  P. Devroey,et al.  Pregnancies after intracytoplasmic injection of single spermatozoon into an oocyte , 1992, The Lancet.

[68]  R. Wall,et al.  Pronuclear microinjection. , 2001, Cloning and stem cells.

[69]  M. Hirabayashi.  Technical development for production of gene-modified laboratory rats. , 2008, The Journal of reproduction and development.

[70]  U. Hengge,et al.  Cytokine gene expression in epidermis with biological effects following injection of naked DNA , 1995, Nature Genetics.

[71]  F. Recillas-Targa.  Multiple strategies for gene transfer, expression, knockdown, and chromatin influence in mammalian cell lines and transgenic animals , 2006, Molecular biotechnology.

[72]  R. Yanagimachi,et al.  Behavior of nuclei of testicular, caput and cauda epididymal spermatozoa injected into hamster eggs. , 1977, Biology of reproduction.

[73]  P. Devroey,et al.  Birth after preimplantation diagnosis of the cystic fibrosis delta F508 mutation by polymerase chain reaction in human embryos resulting from intracytoplasmic sperm injection with epididymal sperm. , 1994, JAMA.

[74]  T. Flotte,et al.  Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. , 1996, Gene therapy.

[75]  U. Müller,et al.  Ten years of gene targeting: targeted mouse mutants, from vector design to phenotype analysis , 1999, Mechanisms of Development.

[76]  J. Pryor,et al.  Comparison of SUZI and ICSI for severe male factor. , 1995, Human reproduction.

[77]  S. Epstein,et al.  Angiogenic-induced enhancement of collateral blood flow to ischemic myocardium by vascular endothelial growth factor in dogs. , 1994, Circulation.

[78]  S. Fishel,et al.  Should ICSI be the treatment of choice for all cases of in-vitro conception? , 2000, Human reproduction.

[79]  J. Contreras,et al.  Improving adenoviral vectors for cancer gene therapy , 1998 .

[80]  David Baltimore,et al.  Germline Transmission and Tissue-Specific Expression of Transgenes Delivered by Lentiviral Vectors , 2002, Science.

[81]  R. Linden,et al.  AAVant-garde gene therapy , 1999, Nature Medicine.

[82]  B. Doe,et al.  Genomic DNA Damage in Mouse Transgenesis1 , 2007, Biology of reproduction.