**Author details**

Kriengkrai Srithanaviboonchai¹,²\*


## **References**


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**5. Conclusion**

182 Selected Topics in Neonatal Care

debate in the years to come.

Kriengkrai Srithanaviboonchai¹,²\*

\*Address all correspondence to: kriengkrai@rihes.org

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1 Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand

2 Research Institute for Health Sciences, Chiang Mai University, Chiang Mai, Thailand

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NMC is as an effective preventive health measure. The medical benefits of NMC outweigh the risks, especially after the effect on HIV prevention has been added. Nevertheless, NMC has not been used to its full potential due to controversies and concerns over the subject. Many issues need to be addressed, if NMC is to be implemented as a public health measure. Implementation of NMC shall vary according to the local context. Parents should be informed about the benefits and risks of NMC where the service is available so that they can decide whether their children should be circumcised. NMC is and will be a health intervention under


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**Section 4**

**Miscellaneous Topics in the Field of Neonatal**

**Health**

**Miscellaneous Topics in the Field of Neonatal Health**

**Chapter 12**

**Provisional chapter**

**Neonatal Gene Therapy for Inherited Disorders**

**Neonatal Gene Therapy for Inherited Disorders**

DOI: 10.5772/intechopen.69218

In spite of developments of neonatal intensive care medicine, it is still difficult or impos‐ sible to treat several inherited genetic disorders using conventional pharmacological methods. Gene therapy is a promising alternate approach for treating a variety of genetic disorders. By the time the patient reaches adulthood, however, it is often too late for effec‐ tive treatment. But in several of these cases, neonatal gene therapy appears potentially useful against inherited disorders that are not obviously treatable through any other methods. This chapter describes the strategy for neonatal gene therapy for inherited disorders and presents preclinical neonatal gene therapy data for two inherited disor‐ ders, metachromatic leukodystrophy and hypophosphatasia. We also discuss the utility, advantages, problems and potential of neonatal gene therapy for inherited disorders.

**Keywords:** neonatal gene therapy, AAV vectors, metachromatic leukodystrophy,

Although there have been significant advances in neonatal intensive care medicine, several neonatal disorders remain major causes of mortality and morbidity. Consequently, there is an urgent need for development of new safe and effective therapies to improve the outcomes of these intractable and devastating neonatal disorders. Gene therapy is an exciting and promis‐ ing approach to treat many diseases for which there are still no effective therapies. To date, more than 2400 clinical trials of gene therapy protocols have been attempted in effort to treat various genetic diseases as well as many types of cancers and infectious diseases (http://www. abedia.com/wiley/continents.php). The results of preclinical studies suggest that neonatal gene therapies represent potentially effective treatments for currently intractable neonatal

> © 2016 The Author(s). Licensee InTech. This chapter is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use, distribution,

© 2018 The Author(s). Licensee IntechOpen. This chapter is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use,

distribution, and reproduction in any medium, provided the original work is properly cited.

and reproduction in any medium, provided the original work is properly cited.

Koichi Miyake, Noriko Miyake and Takashi Shimada

Additional information is available at the end of the chapter

Additional information is available at the end of the chapter

Koichi Miyake, Noriko Miyake and Takashi

http://dx.doi.org/10.5772/intechopen.69218

**Abstract**

hypophosphatasia

**1. Introduction**

Shimada

**Provisional chapter**
