**Abbreviations used**

*Congenital Anomalies in Newborn Infants - Clinical and Etiopathological Perspectives*

gene therapy could also be a definitive treatment for patients.

proportion to the severity of the disease manifestations.

for future clinical applications [53, 54].

**9. Conclusions**

to be addressed.

**Acknowledgements**

**Conflict of interest**

their sincere cooperation and support.

The authors declare no conflict of interest.

trials the duration of bleeding was significantly reduced in number and severity, and also the treated patients not needed blood transfusions and thrombopoietin receptor agonists, platelet counts failed to normal in either trial that it isn't clear. The third trial of WAS gene therapy supported lentiviral vector has recently begun in Boston, USA [52]. At the identical time, other US researchers have developed an alternate lentiviral vector with a stronger WASp expression that's being developed

In line with current studies, it will be acknowledged that gene modification by lentivirus from autologous hematopoietic ancestors can have significant benefits for patients undergoing treatment and be considered as treatment options for WAS. However, more comprehensive studies are needed to ascertain whether this type of

Despite the rarity of WAS, extensive progress has been made in understanding its pathophysiological foundations, but it is still necessary to establish multifaceted management to assess various aspects of the disease. It is worth noting that health care centers have been pioneers in the diagnosis and management of WAS. Significant advances in allogeneic HCT and its valuable long-term results have made it a viable treatment option for most patients with WAS. For severe manifestations, for example, definitive treatment with HCT is recommended. However, even in a clear clinical situation, HCT may not be available due to the patient's geographical location or socioeconomic status, so supportive care measures should be taken promptly. The same is true of WAS cases with milder clinical manifestations; In fact, more emphasis is placed on accepting the potential risks of treatment in

Gene therapy is a potential treatment solution for WAS patients with severe and even mild phenotypes. In this regard, the emergence of advances in the use of gene editing technology creates a cautious optimism. However, the financial and geographical problems for patients with limited access to gene therapy options need

This work has been financed by Intechopen publications and I would like to thank Dr. Hridayesh Prakash as well as dear Ms. Marijana Francetic for closed cooperation and sincere support. I thank Dr. David Buchbinder from Children's Hospital of Orange Country and Dr. Hans D. Ochs from the University of Washington for

I am grateful to my family, Dr. Tooba Ghazanfari from Shahed University, Dr. Farideh Talebi from Shahed University, Dr. Mohammad Mahmoudi from Iran

**190**

**Thanks**

